EP4689135A1 - Promoter - Google Patents

Promoter

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Publication number
EP4689135A1
EP4689135A1 EP24718575.4A EP24718575A EP4689135A1 EP 4689135 A1 EP4689135 A1 EP 4689135A1 EP 24718575 A EP24718575 A EP 24718575A EP 4689135 A1 EP4689135 A1 EP 4689135A1
Authority
EP
European Patent Office
Prior art keywords
promoter
seq
aav vector
transgene
vector
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
EP24718575.4A
Other languages
German (de)
French (fr)
Inventor
Martin MADILL
Kritima GURUNG
Alan GRIFFITH
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Purespring Therapeutics Ltd
Original Assignee
Purespring Therapeutics Ltd
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Filing date
Publication date
Application filed by Purespring Therapeutics Ltd filed Critical Purespring Therapeutics Ltd
Publication of EP4689135A1 publication Critical patent/EP4689135A1/en
Pending legal-status Critical Current

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Definitions

  • the present invention relates to promoters which are capable of driving transgene expression in podocytes.
  • the present invention also relates to vectors comprising said promoters and their use in therapy.
  • BACKGROUND TO THE INVENTION There are many diseases that affect kidney function by attacking the glomerulus.
  • the glomerulus filters approximately 180 litres of plasma each day, and the healthy glomerular filtration barrier has an astonishing ability to retain about 99.9% of large proteins including albumin over our lifetimes without clogging.
  • the glomerular filtration barrier (GFB) comprises 3 main layers: the glomerular endothelial cell, the glomerular basement membrane (GBM) and the podocyte.
  • the GBM is made of a highly cross-linked macromolecular meshwork of type IV collagen, proteoglycans, and laminin. Genetic forms of glomerular disease can be caused by genetic defects in these molecular structures. For example, Alport syndrome is caused by pathogenic variants in the COL4A3, COL4A4 and COL4A5 genes, which result in abnormalities of the collagen IV ⁇ 345 network of basement membranes. Alport syndrome affects approximately 1 in 5,000-10,000 of all individuals in continental Europe and the USA. The condition usually presents during childhood and is associated with a spectrum of phenotypes that include a progressive loss of kidney function, and can include hearing loss and eye abnormalities.
  • GBM-associated diseases include Pierson syndrome and Nail-patella syndrome (Chiang, C.K. and Inagi, R., 2010. Nature Reviews Nephrology, 6(9), p.539).
  • the podocyte has also been implicated as a key cell in the progression of glomerular disease.
  • Podocytes are mesodermally derived cells that are highly specialized and found only in the renal glomerulus. They exhibit unique characteristics such as foot processes and slit diaphragms, which are critical for glomerular filtration.
  • Podocyte-associated genetic glomerular diseases include Nephrotic Syndrome, Frasier syndrome and Denys–Drash syndrome, Schimke immuno-osseous dysplasia, and Epstein and Fechtner syndrome. (Chiang, C.K.
  • glomerular cells such as podocytes
  • promoter sequences which can drive transgene expression in glomerular cells, such as podocytes, are required. Wong et al. (American Journal of Physiology Renal Physiology; 2000; 279(6); F1027-32) described a 1.25-kb DNA fragment from the human nephrin promoter and 5′-flanking region that is capable of directing podocyte-specific expression.
  • This promoter has been used to achieve kidney-specific expression of GFP using an AAV9 vector (Picconi et al.; 2014; Molecular Therapy – Methods & Clinical Development; 1, 14014).
  • vector cargo capacity e.g. AAV cargo capacity
  • non-coding elements such as the nephrin or podocin promotors, WPRE elements and polyadenylation sequence.
  • an AAV vector comprising a transgene for use in therapy, wherein said AAV vector is administered to a subject, wherein the transgene is expressed in podocytes of said subject, and wherein the transgene is operably linked to a 5’-truncated podocin promoter.
  • an AAV vector comprising a transgene in the manufacture of a medicament for therapy, wherein said AAV vector is administered to a subject, wherein the transgene is expressed in podocytes of said subject, and wherein the transgene is operably linked to a 5’-truncated podocin promoter.
  • the therapy is gene therapy.
  • a method of expressing a transgene in podocytes of a subject comprising administering an AAV vector comprising the transgene to said subject, wherein the transgene is expressed in podocytes of said subject, and wherein the transgene is operably linked to a 5’-truncated podocin promoter.
  • the 5’-truncated podocin promoter may be a truncate of a full-length podocin promoter wherein the full-length podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 1.
  • the 5’-truncated podocin promoter has a length of about 1.5 kb or less, about 1.4 kb or less, about 1.3 kb or less, about 1.2 kb or less, about 1.1 kb or less, about 1.0 kb or less, about 0.9 kb or less, about 0.8 kb or less, about 0.7 kb or less, or about 0.6 kb or less.
  • the 5’-truncated podocin promoter has a length of 628 base pairs (bp) or less, 600 bp or less, 550 bp or less, 500 bp or less, 450 bp or less, 400 bp or less, 350 bp or less, 300 bp or less, 250 bp or less, or 200 bp or less.
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to any of SEQ ID NOs: 2 or 35-54.
  • the 5’-truncated podocin promoter has a length of about 628 base pairs (bp).
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 2.
  • the 5’-truncated podocin promoter has a length of about 600 base pairs (bp).
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 36.
  • the 5’-truncated podocin promoter has a length of about 578 base pairs (bp).
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 47.
  • the 5’-truncated podocin promoter has a length of about 550 base pairs (bp).
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 37.
  • the 5’-truncated podocin promoter has a length of about 528 base pairs (bp).
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 48.
  • the 5’-truncated podocin promoter has a length of about 500 base pairs (bp).
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 38.
  • the 5’-truncated podocin promoter has a length of about 478 base pairs (bp).
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 49.
  • the 5’-truncated podocin promoter has a length of about 450 base pairs (bp).
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 39.
  • the 5’-truncated podocin promoter has a length of about 428 base pairs (bp).
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 50.
  • the 5’-truncated podocin promoter has a length of about 400 base pairs (bp).
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 40.
  • the 5’-truncated podocin promoter has a length of about 378 base pairs (bp).
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 51.
  • the 5’-truncated podocin promoter has a length of about 350 base pairs (bp).
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 41.
  • the 5’-truncated podocin promoter has a length of about 328 base pairs (bp).
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 52.
  • the 5’-truncated podocin promoter has a length of about 300 base pairs (bp).
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 42.
  • the 5’-truncated podocin promoter has a length of about 278 base pairs (bp).
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 53.
  • the 5’-truncated podocin promoter has a length of about 250 base pairs (bp).
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 43.
  • the 5’-truncated podocin promoter has a length of about 228 base pairs (bp).
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 54.
  • the 5’-truncated podocin promoter has a length of about 200 base pairs (bp).
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 44.
  • the 5’-truncated podocin promoter has a length of about 150 base pairs (bp).
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 45.
  • the AAV vector used in the present invention is preferably administered to the subject in the form of an AAV vector particle.
  • the AAV vector particle may be encapsidated by AAV3B, LK03, AAV9, ShH10, AAV-DJ, AAV2, AAV6.2, or AAV5 capsid proteins.
  • the AAV vector particle is encapsidated by AAV3B, LK03, or AAV9 capsid proteins. In one embodiment, the AAV vector particle is encapsidated by LK03 capsid proteins. In one embodiment, the AAV vector particle is encapsidated by AAV9 capsid proteins. In one embodiment, the AAV vector particle is not encapsidated by AAV8 capsid proteins or AAV9 capsid proteins. In one embodiment, the AAV vector particle is encapsidated by ShH10, AAV-DJ, AAV2, AAV6.2, or AAV5 capsid proteins.
  • the transgene used in the present invention is a therapeutic transgene.
  • the AAV vector does not comprise a reporter transgene, such as LacZ.
  • the AAV vector does not comprise a nephrotic syndrome-associated transgene, such as NPHS2, ADCK4, ALG1, ARHGAP24, ARGHDIA, CD151, CD2AP, COQ2, COQ6, DGKE, E2F3, EMP2, KANK2, LAGE3, LMNA, LMX1B, MAFB, NUP85, NUP93, NXF5, OSGEP, PAX2, PDSS2, PMM2, PODXL, SCARB2, SGPL1, Smad7, TP53RK, TPRKB, VDR, WDR73, WT1, ZMPSTE24, APOL1, NPHS1, TRPC6, NUP107, NUP133, NUP160, ACTN4, INF2, ANKFY1, ANLN, CRB2, ITGA3, KANK1, KANK4, MAGI2, MYO1E, OCRL, PTPRO, SMARCAL1,
  • the AAV vector does not comprise a vascular endothelial growth factor (VEGFC) transgene. In one embodiment, the AAV vector does not comprise a COL4A3, COL4A4, or COL4A5 transgene. In one embodiment, the AAV vector does not comprise a nucleotide sequence encoding a complement protein, such as CFI, CFH, FHL-1, C1INH, C4BP, MASP2, C3, C5aR1, C5, C5a, CD55, CD35, CD46, CD59, vitronectin, or clusterin.
  • a complement protein such as CFI, CFH, FHL-1, C1INH, C4BP, MASP2, C3, C5aR1, C5, C5a, CD55, CD35, CD46, CD59, vitronectin, or clusterin.
  • the AAV vector does not comprise a NPHS2, ADCK4, ALG1, ARHGAP24, ARGHDIA, CD151, CD2AP, COQ2, COQ6, DGKE, E2F3, EMP2, KANK2, LAGE3, LMNA, LMX1B, MAFB, NUP85, NUP93, NXF5, OSGEP, PAX2, PDSS2, PMM2, PODXL, SCARB2, SGPL1, Smad7, TP53RK, TPRKB, VDR, WDR73, WT1, ZMPSTE24, APOL1, NPHS1, TRPC6, NUP107, NUP133, NUP160, ACTN4, INF2, ANKFY1, ANLN, CRB2, ITGA3, KANK1, KANK4, MAGI2, MYO1E, OCRL, PTPRO, SMARCAL1, SYNPO, TBC1D8B, XPO5, TNS2.
  • the transgene may be operably linked to a Woodchuck hepatitis post-transcriptional regulatory element (WPRE).
  • WPRE Woodchuck hepatitis post-transcriptional regulatory element
  • the transgene may be operably linked to a polyadenylation signal.
  • the transgene may be operably linked to a Kozak sequence.
  • the expression of the transgene in the podocytes of a subject administered the AAV vector defined herein treats or prevents a kidney disease or a glomerular disease in said subject.
  • the subject may have a defective gene corresponding to said transgene.
  • the subject is a human subject.
  • the subject is suffering from or is at risk of a kidney disease or a glomerular disease.
  • the AAV vector may be administered in the form of a pharmaceutical composition comprising a pharmaceutically acceptable carrier, diluent or excipient.
  • the AAV vector may be administered systemically and/or by intravenous injection.
  • the AAV vector may be administered locally and/or by injection into the renal artery.
  • the AAV vector may be administered in a dose of from about 1x10 6 vg/kg to about 1x10 14 vg/kg, from about 1x10 6 vg/kg to about 1x10 13 vg/kg, or from about 1x10 9 vg/kg to about 1x10 12 vg/kg.
  • the administration of the AAV vector may result in the transgene being specifically expressed in podocytes.
  • the transgene is expressed in 5% or more, 10% or more, 15% or more, 20% or more, 30% or more or 40% or more podocytes in at least one of the subject’s kidneys.
  • an AAV vector comprising a therapeutic transgene, wherein the transgene is operably linked to a 5’-truncated podocin promoter as defined herein, and wherein the AAV vector does not comprise: (i) a nephrotic syndrome-associated transgene, such as NPHS2, ADCK4, ALG1, ARHGAP24, ARGHDIA, CD151, CD2AP, COQ2, COQ6, DGKE, E2F3, EMP2, KANK2, LAGE3, LMNA, LMX1B, MAFB, NUP85, NUP93, NXF5, OSGEP, PAX2, PDSS2, PMM2, PODXL, SCARB2, SGPL1, Smad7, TP53RK, TPRKB, VDR, WDR73, WT1, ZMPSTE24, APOL1, NPHS1, TRPC6, NUP107, NUP133, NUP160, ACTN4, INF2, ANKFY1, ANLN, C
  • an AAV vector particle encapsidated by ShH10, AAV-DJ, AAV2, AAV6.2, or AAV5 capsid proteins and comprising a therapeutic transgene, wherein the transgene is operably linked to a 5’-truncated podocin promoter as defined herein.
  • a cell comprising the AAV vector defined herein.
  • a pharmaceutical composition comprising the AAV vector or the cell defined herein.
  • a promoter consisting of a nucleotide sequence having at least 97.8% identity to SEQ ID NO: 2.
  • the promoter consists of a nucleotide sequence having at least 97.9%, at least 98.0%, at least 98.1%, at least 98.2%, at least 98.3%, at least 98.4%, at least 98.5%, at least 98.6%, at least 98.7%, at least 98.8%, at least 98.9%, at least 99.0%, at least 99.1%, at least 99.2%, at least 99.3%, at least 99.4%, at least 99.5%, at least 99.6%, at least 99.7%, at least 99.8%, or at least 99.9% identity to SEQ ID NO: 2.
  • the promoter consists of the nucleotide sequence of SEQ ID NO: 2.
  • a 5’-truncated podocin promoter having a length of 600bp or less.
  • the 5’-truncated podocin promoter has a length of 550bp or less, 500bp or less, 450bp or less, 400bp or less, 350 bp or less, 300 bp or less, 250 bp or less, or 200 bp or less.
  • the 5’-truncated podocin promoter may be a truncate of a full-length podocin promoter wherein the full-length podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 1.
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to any of SEQ ID NOs: 36-54.
  • a promoter which consists of a nucleotide sequence having at least 90% identity to any of SEQ ID NOs: 36-54.
  • the promoter consists of a nucleotide sequence having at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to any of SEQ ID NOs: 36-54.
  • the promoter consists of a nucleotide sequence having at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to any of SEQ ID NOs: 37-46 or 47-54, any of SEQ ID NOs: 37-46 or 48-54, any of SEQ ID NOs: 38-46 or 48-54, any of SEQ ID NOs: 38- 46 or 49-54, any of SEQ ID NOs: 39-46 or 49-54, any of SEQ ID NOs: 39-46 or 50-54, any of SEQ ID NOs: 40-46 or 50-54, any of SEQ ID NOs: 40-46 or 50-54, any of SEQ ID NOs: 40-46 or 51-54, any of SEQ ID NOs: 41- 46 or 51-54, any of SEQ ID NOs: 41-46 or 52-54, any of SEQ ID NOs: 42-
  • the promoter consists of a nucleotide sequence having at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to any of SEQ ID NOs: 38, 40, 41, 42, 44, 47, 48, 51 or 53.
  • the promoter consists of a nucleotide sequence having at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to any of SEQ ID NOs: 40, 47 or 53.
  • a polynucleotide comprising the promoter as defined herein.
  • the promoter is operably linked to a transgene.
  • the transgene is operably linked to one or more further regulatory elements, such as a post- transcriptional regulatory element and/or a polyadenylation sequence.
  • a vector comprising the polynucleotide as defined herein.
  • the vector is capable of transducing podocytes, optionally wherein the vector is capable of specifically transducing podocytes.
  • the vector is a viral vector, such as an adeno-associated virus (AAV) vector, a lentiviral vector, a retroviral vector, an adenoviral vector, an adeno-associated viral vector, a herpes simplex viral vector, an alphaviral vector, a flaviviral vector, a rhabdoviral vector, a measles viral vector, a Newcastle disease viral vector, a poxviral vector, or a picornaviral vector, preferably wherein the vector is an AAV vector.
  • the viral vector is in the form of a viral vector particle, preferably wherein the viral vector is in the form of an AAV vector particle.
  • the vector is the form of an AAV vector particle encapsidated by AAV3B, LK03, AAV9, ShH10, AAV-DJ, AAV2, AAV6.2, or AAV5 capsid proteins.
  • a cell comprising the polynucleotide, or the vector as defined herein.
  • a pharmaceutical composition comprising the polynucleotide, the vector, or the cell as defined herein.
  • FIG. 1 Transfection of HEK293T cells with FL (full-length) and 628bp promoters driving GFP expression.
  • Figure 2 Transfection of mouse podocytes with FL (full-length) and 628bp promoters driving GFP expression.
  • Figure 3 Transfection of HEK293T cells with FL (full-length) and 628bp promoters driving Luciferase expression compared to non-transfected (NT) cells.
  • Figure 4 Schematic of constructs used in Examples 1-3.
  • B NPSH2(628)-eGFP construct used in Examples 1 and 2.
  • C NPHS2(FL)-Luciferase-GFP construct used in Example 3.
  • C NPHS2(628)-Luciferase-GFP construct used in Example 3.
  • AD293 cells were transfected with plasmids comprising a luciferase transgene under the control of truncated podocin promoters having lengths of from 150 bp to 600 bp (PS0576 to PS0593).
  • Luciferase activity is compared to non-transfected cells and 628bp promoter (PS0471).
  • PS0471 628bp promoter
  • Promoters A “promoter” is used herein according to its typical meaning to refer to a sequence of DNA which regulates and/or initiates transcription of RNA from a DNA sequence.
  • the promoter is operable in mammalian cells, e.g. human cells.
  • the promoter may be operable in kidney cells.
  • the promoter of the invention may be capable of driving transgene expression in the kidney.
  • kidney cells in which the promoter may be operable include, but are not limited to, glomerular cells.
  • the promoter of the invention may be capable of driving transgene expression in the glomerulus.
  • the mature glomerulus contains four cell types: Parietal epithelial cells that form Bowman's capsule, podocytes that cover the outermost layer of the glomerular filtration barrier, glycocalyx-coated fenestrated endothelial cells that are in direct contact with blood, and mesangial cells that sit between the capillary loops (Vaughan, M.R. and Quaggin, S.E., 2008. Journal of the American Society of Nephrology, 19(1), pp.24-33).
  • the promoter may be operable in a podocyte cell.
  • the promoter of the invention may be capable of driving transgene expression in podocytes.
  • the promoter of the invention may be a tissue-specific promoter.
  • tissue-specific promoter is a promoter which preferentially facilitates expression of a transgene in a specific type of cells or tissue.
  • a tissue-specific promoter may facilitate higher expression of a transgene in one cell-type as compared to other cell-types.
  • a tissue-specific promoter may be a promoter which facilitates transgene expression levels at least 10% higher, at least 20% higher, at least 30% higher, at least 40% higher, at least 50% higher, at least 100% higher, at least 200% higher, at least 300% higher, at least 400% higher, at least 500% higher, or at least 1000% higher in one cell-type as compared to expression levels in other cell-types.
  • the promoter is a kidney-specific promoter.
  • the promoter is a podocyte-specific promoter.
  • Transgene expression may be measured by any suitable method known in the art. For example by measuring the expression of a reporter transgene, e.g. Green fluorescent protein (GFP), operably linked to the promoter, wherein expression of the reporter transgene correlates with the ability of the promoter to facilitate expression of a gene.
  • a reporter transgene e.g. Green fluorescent protein (GFP)
  • GFP Green fluorescent protein
  • Expression of the reporter transgene, e.g. GFP may be determined by any suitable method e.g. FACS.
  • a kidney-specific promoter may facilitate higher expression of a reporter transgene in kidney cells compared to other cell types (e.g.
  • a podocyte-specific promoter may facilitate higher expression of a reporter transgene in conditionally immortalised podocytes compared to other cell-types.
  • Suitable podocyte cell lines will be well known to those of skill in the art, for example CIHP-1.
  • Methods to generate immortalized podocytes will be well known to those of skill in the art. Suitable methods are described in Ni, L., et al., 2012. Nephrology, 17(6), pp.525-531.
  • the 5’-truncated podocin promoter used in the invention is a truncate of a full-length podocin promoter.
  • the full-length podocin promoter sequence has at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 1 shown below.
  • a further full-length podocin promoter is shown below as SEQ ID NO: 34.
  • the full-length podocin promoter sequence has at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 34 shown below.
  • the 5’-truncated podocin promoter has a length of 628 bp or less. In one embodiment the 5'-truncated podocin promoter has a length of 578-678 bp, 583-673 bp, 588- 668 bp, 593-663 bp, 598-658 bp, 603-653 bp, 608-648 bp, 613-643 bp, 618-638 bp, or 623- 633 bp. In one embodiment the 5’-truncated podocin promoter has a length of 628 bp.
  • the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 2 shown below.
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 35 shown below.
  • the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least at least 97.0%, at least 97.1%, at least 97.2%, at least 97.3%, at least 97.4%, at least 97.5%, at least 97.6%, at least 97.7%, at least 97.8%, at least 97.9%, at least 98.0%, at least 98.1%, at least 98.2%, at least 98.3%, at least 98.4%, at least 98.5%, at least 98.6%, at least 98.7%, at least 98.8%, at least 98.9%, at least 99.0%, at least 99.1%, at least 99.2%, at least 99.3%, at least 99.4%, at least 99.5%, at least 99.6%, at least 99.7%, at least 99.8%, at least 99.9%, or 100.0% identity to SEQ ID NO: 35.
  • the 5’-truncated podocin promoter has a length of 550-650 bp, 555-645 bp, 560- 640 bp, 565-635 bp, 570-630 bp, 575-625 bp, 580-620 bp, 585-615 bp, 590-610 bp, or 595- 605 bp. In one embodiment the 5’-truncated podocin promoter has a length of 600 bp.
  • the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 36 shown below.
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 36 shown below.
  • the 5’-truncated podocin promoter has a length of 528-628 bp, 533-623 bp, 538- 618 bp, 543-613 bp, 548-608 bp, 553-603 bp, 558-598 bp, 563-593 bp, 568-588 bp, or 573- 583 bp. In one embodiment the 5’-truncated podocin promoter has a length of 578 bp.
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 47 shown below.
  • the 5’-truncated podocin promoter has a length of 500-600 bp, 505-595 bp, 510- 590 bp, 515-585 bp, 520-580 bp, 525-575 bp, 530-570 bp, 535-565 bp, 540-560 bp, or 545- 555 bp. In one embodiment the 5’-truncated podocin promoter has a length of 550 bp.
  • the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 37 shown below.
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 37 shown below.
  • the 5'-truncated podocin promoter has a length of 478-578 bp, 483-573 bp, 488- 568 bp, 493-563 bp, 498-558 bp, 503-553 bp, 508-548 bp, 513-543 bp, 518-538 bp, or 523- 533 bp. In one embodiment the 5’-truncated podocin promoter has a length of 528 bp.
  • the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 48 shown below.
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 48 shown below.
  • the 5'-truncated podocin promoter has a length of 450-550 bp, 455-545 bp, 460- 540 bp, 465-535 bp, 470-530 bp, 475-525 bp, 480-520 bp, 485-515 bp, 490-510 bp, or 495- 505 bp. In one embodiment the 5’-truncated podocin promoter has a length of 500 bp.
  • the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 38 shown below.
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 38 shown below.
  • the 5'-truncated podocin promoter has a length of 428-528 bp, 433-523 bp, 438- 518 bp, 443-513 bp, 448-508 bp, 453-503 bp, 458-498 bp, 463-493 bp, 468-488 bp, or 473- 483 bp. In one embodiment the 5’-truncated podocin promoter has a length of 478 bp.
  • the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 49 shown below.
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 49 shown below.
  • the 5'-truncated podocin promoter has a length of 400-500 bp, 405-495 bp, 410- 490 bp, 415-485 bp, 420-480 bp, 425-475 bp, 430-470 bp, 435-465 bp, 440-460 bp, or 445- 455 bp. In one embodiment the 5’-truncated podocin promoter has a length of 450 bp.
  • the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 39 shown below.
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 39 shown below.
  • the 5'-truncated podocin promoter has a length of 378-478 bp, 383-473 bp, 388- 468 bp, 393-463 bp, 398-458 bp, 403-453 bp, 408-448 bp, 413-443 bp, 418-438 bp, or 423- 433 bp. In one embodiment the 5’-truncated podocin promoter has a length of 428 bp.
  • the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 50 shown below.
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 50 shown below.
  • the 5'-truncated podocin promoter has a length of 350-450 bp, 355-445 bp, 360- 440 bp, 365-435 bp, 370-430 bp, 375-425 bp, 380-420 bp, 385-415 bp, 390-410 bp, or 395- 405 bp. In one embodiment the 5’-truncated podocin promoter has a length of 400 bp.
  • the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 40 shown below.
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 40 shown below.
  • the 5'-truncated podocin promoter has a length of 328-428 bp, 333-423 bp, 338- 418 bp, 343-413 bp, 348-408 bp, 353-403 bp, 358-398 bp, 363-393 bp, 368-388 bp, or 373- 383 bp. In one embodiment the 5’-truncated podocin promoter has a length of 378 bp.
  • the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 51 shown below.
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 51 shown below.
  • the 5'-truncated podocin promoter has a length of 300-400 bp, 305-395 bp, 310- 390 bp, 315-385 bp, 320-380 bp, 325-375 bp, 330-370 bp, 335-365 bp, 340-360 bp, or 345- 355 bp. In one embodiment the 5’-truncated podocin promoter has a length of 350 bp.
  • the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 41 shown below.
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 41 shown below.
  • the 5'-truncated podocin promoter has a length of 278-378 bp, 283-373 bp, 288- 368 bp, 293-363 bp, 298-358 bp, 303-353 bp, 308-348 bp, 313-343 bp, 318-338 bp, or 323- 333 bp. In one embodiment the 5’-truncated podocin promoter has a length of 328 bp.
  • the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 52 shown below.
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 52 shown below.
  • the 5’-truncated podocin promoter has a length of 300 bp or less.
  • the 5'-truncated podocin promoter has a length of 250-350 bp, 255-345 bp, 260- 340 bp, 265-335 bp, 270-330 bp, 275-325 bp, 280-320 bp, 285-315 bp, 290-310 bp, or 295- 305 bp. In one embodiment the 5’-truncated podocin promoter has a length of 300 bp.
  • the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 42 shown below.
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 42 shown below.
  • the 5'-truncated podocin promoter has a length of 228-328 bp, 233-323 bp, 238- 318 bp, 243-313 bp, 248-308 bp, 253-303 bp, 258-298 bp, 263-293 bp, 268-288 bp, or 273- 283 bp. In one embodiment the 5’-truncated podocin promoter has a length of 278 bp.
  • the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 53 shown below.
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 53 shown below.
  • the 5’-truncated podocin promoter has a length of 250 bp or less.
  • the 5'-truncated podocin promoter has a length of 200-300 bp, 205-295 bp, 210- 290 bp, 215-285 bp, 220-280 bp, 225-275 bp, 230-270 bp, 235-265 bp, 240-260 bp, or 245- 255 bp. In one embodiment the 5’-truncated podocin promoter has a length of 250 bp.
  • the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 43 shown below.
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 43 shown below.
  • the 5’-truncated podocin promoter has a length of 228 bp or less.
  • the 5'-truncated podocin promoter has a length of 178-278 bp, 183-273 bp, 188- 268 bp, 193-263 bp, 198-258 bp, 203-253 bp, 208-248 bp, 213-243 bp, 218-238 bp, or 223- 233 bp. In one embodiment the 5’-truncated podocin promoter has a length of 228 bp.
  • the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 54 shown below.
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 54 shown below.
  • the 5’-truncated podocin promoter has a length of 200 bp or less.
  • the 5'-truncated podocin promoter has a length of 150-250 bp, 155-245 bp, 160- 240 bp, 165-235 bp, 170-230 bp, 175-225 bp, 180-220 bp, 185-215 bp, 190-210 bp, or 195- 205 bp. In one embodiment the 5’-truncated podocin promoter has a length of 200 bp.
  • the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 44 shown below.
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 44 shown below.
  • the 5’-truncated podocin promoter has a length of 150 bp or less.
  • the 5'-truncated podocin promoter has a length of 100-200 bp, 105-195 bp, 110- 190 bp, 115-185 bp, 120-180 bp, 125-175 bp, 130-170 bp, 135-165 bp, 140-160 bp, or 145- 155 bp. In one embodiment the 5’-truncated podocin promoter has a length of 150 bp.
  • the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 45 shown below.
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 45 shown below.
  • the 5’-truncated podocin promoter has a length of 100 bp or less. In one embodiment the 5’-truncated podocin promoter has a length of 100 bp.
  • the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 46 shown below.
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 46 shown below.
  • the 5’-truncated podocin promoter may have a length of about 100 bp or more, 150 bp or more, 200 bp or more, 250 bp or more, 300 bp or more, 350 bp or more, 400 bp or more, 450 bp or more, 500 bp or more, 550 bp or more, or 600 bp or more. In one embodiment, the 5’- truncated podocin promoter has a length of about 150 bp or more.
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to any of SEQ ID NOs: 2 or 35-54.
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to any of SEQ ID NOs: 36-54.
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to any of SEQ ID NOs: 36-46.
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to any of SEQ ID NOs: 47-54.
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to any of SEQ ID NOs: 38, 40, 41, 42, 44, 47, 48, 51, 53.
  • the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to any of SEQ ID NOs: 40, 47, 53.
  • Polynucleotides In one aspect, the present invention provides a polynucleotide comprising the promoter described herein. Polynucleotides of the invention may comprise DNA or RNA, preferably DNA. They may be single-stranded or double-stranded.
  • Polynucleotides such as DNA polynucleotides may be produced recombinant, synthetically or by any means available to those of skill in the art. They may also be cloned by standard techniques.
  • the polynucleotide may be an isolated polynucleotide. Longer polynucleotides will generally be produced using recombinant means, for example using polymerase chain reaction (PCR) cloning techniques. This will involve making a pair of primers (e.g.
  • the primers may be designed to contain suitable restriction enzyme recognition sites so that the amplified DNA can be cloned into a suitable vector.
  • the polynucleotides may be modified by any method available in the art. Such modifications may be carried out in order to enhance the in vivo activity or lifespan of the polynucleotides of the invention.
  • the promoter referred to herein may be operably linked to one or more transgenes.
  • the transgene is a therapeutic transgene.
  • the transgene may encode any polypeptide of interest.
  • the transgene sequence man encode any polypeptide associated with a kidney disease such as a glomerular disease.
  • the transgene may encode a polypeptide involved in a GBM-associated genetic glomerular disease.
  • the transgene may encode a polypeptide involved in a podocyte-associated genetic glomerular disease.
  • the transgene may encode a COL4A3, COL4A4, COL4A5, NPHS2, CFH, CFL, CFHL1, C1INH, C4BP, MASP2, C3, C5aR1, C5, C5a, CD55, CD35, CD46, CD59, vitronectin, clusterin, ADCK4, ALG1, ARHGAP24, ARGHDIA, CD151, CD2AP, COQ2, COQ6, DGKE, E2F3, EMP2, KANK2, LAGE3, LMNA, LMX1B, MAF B, NUP85, NUP93, NXF5, OSGEP, PAX2, PDSS2, PMM2, PODXL, SCARB2, SGPL1, Smad7, TP53RK, TPRKB, VDR, WDR73, WT1, ZMPSTE24, APOL1, NPHS1, TRPC6, NUP107, NUP133, NUP160, ACTN4, INF2, ANKFY1, ANLN, C
  • polynucleotide For a protein-coding polynucleotide, it will be understood by a skilled person that numerous different polynucleotides can encode the same polypeptide as a result of the degeneracy of the genetic code. In addition, it is to be understood that skilled persons may, using routine techniques, make nucleotide substitutions that do not affect the polypeptide sequence encoded by the polynucleotides of the invention to reflect the codon usage of any particular host organism in which the polypeptides of the invention are to be expressed.
  • the transgene may be codon-optimised. Different cells differ in their usage of particular codons. This codon bias corresponds to a bias in the relative abundance of particular tRNAs in the cell type.
  • codons in the sequence By altering the codons in the sequence so that they are tailored to match with the relative abundance of corresponding tRNAs, it is possible to increase expression. Similarly, it is possible to decrease expression by deliberately choosing codons for which the corresponding tRNAs are known to be rare in the particular cell type. Thus, an additional degree of translational control is available. Codon usage tables are known in the art for mammalian cells (e.g. humans), as well as for a variety of other organisms.
  • the transgenes disclosed herein may comprise or lack stop codons at their 3’ end. Thus, the present disclosure encompasses the SEQ ID NOs disclosed herein with the stop codons present or absent.
  • the transgene may encode a COL4A3, COL4A4 or COL4A5 polypeptide, or a fragment or derivative thereof.
  • COL4A3, COL4A4 and COL4A5 proteins are approximately 170-185 kDa homologous polypeptides containing collagenous Gly-X-Y repeat sequences frequently interrupted by non- collagenous sequences and forming a triple helix repeat. Each polypeptide also contains a large globular non-collagenous domain at the carboxyl-terminal end.
  • Alport syndrome is caused by pathogenic variants in the COL4A3, COL4A4 and COL4A5 genes, which result in abnormalities of the collagen IV ⁇ 345 network of basement membranes.
  • the COL4A3, COL4A4 or COL4A5 polypeptide or a fragment or derivative thereof may be capable of forming a collagen IV ⁇ 345 network.
  • Approximately 200-300 amino acids may be removed from each of the COL4A3, COL4A4 and COL4A5 polypeptides to produce a truncated transgene suitable for a mini-gene approach.
  • the amino acids may be removed from the triple helix repeat.
  • the amino acids are not removed from the non-collagenous region.
  • the COL4A3, COL4A4 and COL4A5 polypeptides are full-length polypeptides.
  • the COL4A3, COL4A4 or COL4A5 polypeptide is human.
  • An example human COL4A3 is the COL4A3 having the UniProtKB accession number Q01955.
  • An example human COL4A4 is the COL4A4 having the UniProtKB accession number P53420.
  • An example human COL4A5 is the COL4A5 having the UniProtKB accession number P29400.
  • the variant may be at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99% identical to SEQ ID NO: 4.
  • the COL4A5 peptide may comprise or consist of the polypeptide sequence shown as SEQ ID NO: 5, or a variant which is at least 70% identical to SEQ ID NO: 5.
  • the variant may be at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99% identical to SEQ ID NO: 5.
  • COL4A3 amino acid sequence – Uniprot reference Q01955 (SEQ ID NO: 3) MSARTAPRPQVLLLPLLLVLLAAAPAASKGCVCKDKGQCFCDGAKGEKGEKGFPGPPGSPGQKGFTGP EGLPGPQGPKGFPGLPGLTGSKGVRGISGLPGFSGSPGLPGTPGNTGPYGLVGVPGCSGSKGEQGFPG LPGTLGYPGIPGAAGLKGQKGAPAKEEDIELDAKGDPGLPGAPGPQGLPGPPGFPGPVGPPGPPGFFG FPGAMGPRGPKGHMGERVIGHKGERGVKGLTGPPGPPGTVIVTLTGPDNRTDLKGEKGDKGAMGEPGP PGPSGLPGESYGSEKGAPGDPGLQGKPGKDGVPGFPGSEGVKGNRGFPGLMGEDGIKGQKGDIGPPGF RGPTEYYDTYQEKGDEGTPGPPGPRGARGPQGPSGPPGVPGSPGSSRPGLRGAPGWPGLKGSKGERGR PGKDA
  • transgene encoding COL4A4 is provided in NM_000092.5.
  • An example transgene encoding COL4A5 is provided in NM_000495.5.
  • the COL4A3 transgene may comprise or consist of the polynucleotide sequence shown as SEQ ID NO: 6, or a variant which is at least 70% identical to SEQ ID NO: 6.
  • the variant may be at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99% identical to SEQ ID NO: 6.
  • the COL4A4 transgene may comprise or consist of the polynucleotide sequence shown as SEQ ID NO: 7, or a variant which is at least 70% identical to SEQ ID NO: 7.
  • the variant may be at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99% identical to SEQ ID NO: 7.
  • the COL4A5 transgene may comprise or consist of the polynucleotide sequence shown as SEQ ID NO: 8, or a variant which is at least 70% identical to SEQ ID NO: 8.
  • the variant may be at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99% identical to SEQ ID NO: 8.
  • Exemplary COL4A3 transgene sequence (SEQ ID NO: 6) atgagcgcccggaccgcccccaggccgcaggtgctcctgctgctgctggtgctcctggcggcggc ggc ggc ggc ggc ggc ggc ggc ggc ggc ggc ggc ggcgcag gggagaagggggagaagggctttctgtgacggggccaaag gggagaagggggagaagggctttcctggaccccccggttctctggccagaaaggattcacaggtcct gaaggcttgctgg
  • the COL4A3, COL4A4 or COL4A5 transgene may comprise a protein tag, such as a hemagglutinin (HA) tag.
  • HA hemagglutinin
  • the protein tag can facilitate detection, isolation, and purification of the transgene.
  • Other suitable protein tags may include Myc tags, polyhistidine tags and flag tags.
  • Nephrotic syndrome (NS)-associated transgene The transgene may comprise or consist of a NS-associated gene.
  • Nephrotic syndrome (NS) is a chronic kidney disease characterized by significant proteinuria, hypoalbuminemia, oedema and hyperlipidemia.
  • the NS-associated transgene may be a gene associated with a monogenic form of NS and expressed in podocytes.
  • Suitable NS-associated transgenes include NPHS2, ADCK4, ALG1, ARHGAP24, ARGHDIA, CD151, CD2AP, COQ2, COQ6, DGKE, E2F3, EMP2, KANK2, LAGE3, LMNA, LMX1B, MAFB, NUP85, NUP93, NXF5, OSGEP, PAX2, PDSS2, PMM2, PODXL, SCARB2, SGPL1, Smad7, TP53RK, TPRKB, VDR, WDR73, WT1, ZMPSTE24, APOL1, NPHS1, TRPC6, NUP107, NUP133, NUP160, ACTN4, INF2, ANKFY1, ANLN, CRB2, ITGA3, KANK1, KANK4, MAGI2, MYO1E, OCRL, PTPRO, SMARCAL1, SYNPO, T
  • NPHS2 The transgene may encode NPHS2, or a fragment and/or variant thereof.
  • NPHS2 is the abbreviated name of the polypeptide encoded by the NPHS2 gene and is also known as podocin.
  • NPHS2 is a 42kDa hairpin like membrane-associated podocyte-specific protein that is a key component of the protein complex at the slit diaphragm; the cell-cell junction between adjacent podocyte foot processes. It localises to lipid rafts and interacts with other important slit diaphragm proteins like nephrin, CD2AP and TRPC6. It is essential in the maintenance of the slit diaphragm, and consequently the integrity of the glomerular filtration barrier.
  • a fragment and/or variant of NPHS2 may retain NPHS2 function.
  • a fragment and/or variant of podocin may regulate glomerular permeability.
  • a fragment and/or variant of NPHS2 may have the same or similar function to NPHS2, e.g. may have at least 40%, at least 50%, at least 60%, at least 70%, at least 80%, at least 90%, at least 95%, or at least 100% of the function of NPHS2.
  • a person skilled in the art would be able to generate fragments and/or variants using conservative substitutions, based on the known structural and functional features of NPHS2 (see e.g. Tabassum, A., et al., 2014.
  • NPHS2 a fragment and/or variant of NPHS2 comprises a transmembrane domain, with two cytoplasmic domains at the N- and C-terminus.
  • the NPHS2 gene is conserved in chimpanzee, Rhesus monkey, dog, cow, mouse, and rat.
  • the NPHS2 may be a human NPHS2.
  • the NPHS2 may comprise or consist of a polypeptide sequence of UniProtKB accession Q9NP85, or a fragment and/or variant thereof.
  • the NPHS2 polypeptide comprises or consists of an amino acid sequence which is at least 70% identical to SEQ ID NO: 9 or a fragment thereof.
  • the NPHS2 polypeptide comprises or consists of an amino acid sequence which is at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 9 or a fragment thereof.
  • the NPHS2 polypeptide comprises or consists of SEQ ID NO: 9 or a fragment thereof.
  • NPHS2amino acid sequence SEQ ID NO: 9
  • the NPHS2 transgene comprises or consists of a nucleic acid sequence (SEQ ID NO: 9)
  • the NPHS2 transgene comprises or consists of a nucleic acid sequence (SEQ ID NO: 9)
  • the NPHS2 transgene comprises or consists of a nucleic acid sequence (SEQ ID NO: 9)
  • the NPHS2 transgene comprises or consists of a nucleotide sequence which is at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 10 or a fragment thereof.
  • the NPHS2 transgene comprises or consists of the nucleotide sequence SEQ ID NO: 10 or a fragment thereof.
  • the NPHS2 transgene comprises or consists of a nucleotide sequence which is at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 11 or a fragment thereof.
  • the NPHS2 transgene comprises or consists of the nucleotide sequence SEQ ID NO: 11 or a fragment thereof.
  • VEGFC is a lymphangiogenic growth factor, which is known to signal via two receptors, VEGFR-3 (Flt4) and VEGFR-2 (Flk4).
  • VEGFC is produced by cells in a prepropeptide form, which dimerises before being cleaved into a tetramer.
  • the VEGFC transgene may comprise a polynucleotide encoding any form of VEGFC, such as the prepropeptide form, the tetramer form, the intermediate form, or the fully processed mature VEGFC. If desired, polynucleotides encoding different forms of VEGFC polypeptides may be used in any combination.
  • the VEGFC transgene comprises a polynucleotide encoding one or more polypeptides having VEGFC biological activity, i.e., peptides that can bind to and activate VEGFR-2 and/or VEGRF-3. More preferably, the VEGFC transgene comprises a polynucleotide encoding a polypeptide comprising the VEGFC homology domain and having VEGFC biological activity, i.e., a polypeptide that can bind to and activate VEGFR-2 and/or VEGRF-3. Further details of suitable VEGFC polynucleotides and polypeptides include those described in WO 2015/022447 and US 2014/0087002.
  • the VEGFC polynucleotide may comprise the VEGFC open reading frame (ORF) sequence of SEQ ID NO: 12.
  • the VEGFC polynucleotide may comprise a nucleic acid sequence which has at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, or at least 99% sequence identity to the VEGFC ORF sequence of SEQ ID NO: 12.
  • the variant sequence may encode a VEGFC polypeptide that has retained the capability to bind and activate VEGFR-2 and VEGFR-3.
  • VEGFC polynucleotide ATGCACTTGCTGGGCTTCTTCTCTGTGGCGTGTTCTCTGCTCGCCGCTGCGCTGCTCCCGGGTCCTCG CGAGGCGCCCGCCGCCGCCGCCGCCTTCGAGTCCGGACTCGACCTCTCGGACGCGGAGCCCGACGCGG GCGAGGCCACGGCTTATGCAAGCAAAGATCTGGAGGAGCAGTTACGGTCTGTGTCCAGTGTAGATGAA CTCATGACTGTACTCTACCCAGAATATTGGAAAATGTACAAGTGTCAGCTAAGGAAAGGAGGCTGGCA ACATAACAGAGAACAGGCCAACCTCAACTCAAGGACAGAAGACTATAAAATTTGCTGCAGCACATT ATAATACAGAGATCTTGAAAAGTATTGATAATGAGTGGAGAAAGACTCAATGCATGCCACGGGAGGTG TGTATAGATGTGGGGAAGGAGTTTGGAGTCGCGACAAACACCTTCTTTAAACCTCCATGTGTGTCCGT CTACA
  • a “complement protein” is a protein which is part of the complement system.
  • the complement system also known as complement cascade, is a central part of the innate immunity that serves as a first line of defence against foreign and altered host cells.
  • the complement system is composed of plasma proteins produced mainly by the liver or membrane proteins expressed on cell surface. Complement operates in plasma, in tissues, or within cells. Complement proteins collaborate as a cascade to opsonize pathogens and induce a series of inflammatory responses helping immune cells to fight infection and maintain homeostasis (Merle, N.S., et al., 2015. Frontiers in immunology, 6, 262). There are three pathways of complement activation: the classical, the alternative, and the lectin pathways.
  • the complement protein is selected from the list consisting of CFI, CFH, FHL-1, C1INH, C4BP, MASP2, C3, C5aR1, C5, C5a, CD55, CD35, CD46, CD59, vitronectin, and clusterin, or fragments and/or variants thereof.
  • the transgene may encode an inhibitor of the complement system, or a fragment and/or variant thereof.
  • an “inhibitor of the complement system” or “complement inhibitor” is a protein which prevents activation of the complement system. Complement is tightly controlled by these inhibitors, which naturally protect self-cells and tissues from unwanted complement activation. Complement inhibitors can regulate complement activation in different stages of the classical, lectin, and alternative pathways.
  • the complement inhibitor is a naturally- occurring complement inhibitor, or a fragment and/or variant thereof.
  • the inhibitor of the complement system is an inhibitor of the complement system in humans. Complement inhibitors are grouped into two categories: soluble inhibitors and membrane- bound inhibitors.
  • the inhibitor of the complement system is a soluble complement inhibitor.
  • Soluble complement inhibitors include C1 inhibitor (C1INH), complement factor I (CFI), complement factor H (CFH), complement factor H-like protein 1 (FHL-1), C4 binding protein (C4BP), clusterin and vitronectin.
  • Membrane-bound regulators include CD46, CD55, CD59, CD35 and CUB and Sushi multiple domain 1 (CSMD1).
  • the inhibitor of the complement system may be selected from: CFI, CFH, FHL-1, C1INH, C4BP, CD46, CD55, CD59, CD35, vitronectin, clusterin, and CSMD1, or fragments and/or variants thereof.
  • the inhibitor of the complement system is selected from: CFI, CFH, and FHL-1, or fragments and/or variants thereof.
  • CFI The transgene may encode CFI, or a fragment and/or variant thereof.
  • Complement factor I is a trypsin-like serine protease that inhibits the complement system by cleaving three peptide bonds in the alpha-chain of C3b and two bonds in the alpha-chain of C4b thereby inactivating these proteins.
  • CFI is a glycoprotein heterodimer consisting of a disulfide linked heavy chain and light chain.
  • the heavy chain has four domains: an FI membrane attack complex (FIMAC) domain, CD5 domain, and low-density lipoprotein receptor 1 and 2 (LDLr1 and LDLr2) domains.
  • FIMAC FI membrane attack complex
  • CD5 CD5 domain
  • LDLr1 and LDLr2 low-density lipoprotein receptor 1 and 2 domains.
  • the heavy chain plays an inhibitory role in maintaining the enzyme inactive until it meets the complex formed by the substrate (either C3b or C4b) and a cofactor protein (Factor H, C4b-binding protein, complement receptor 1, and membrane cofactor protein).
  • the enzyme Upon binding of the enzyme to the substrate:cofactor complex, the heavy:light chain interface is disrupted, and the enzyme activated by allostery.
  • the light chain contains only the serine protease domain.
  • This domain contains the catalytic triad His-362, Asp-411, and Ser-507, which is responsible for specific cleavage of C3b and C4b.
  • the CFI or a fragment and/or variant thereof may be capable of cleaving C3b into iC3b and/or may be capable of cleaving iC3b into C3d,g.
  • the fragment and/or variant of CFI may retain at least 50%, at least 60%, at least 70%, at least 80%, at least 90%, at least 95% or 100% of the C3b-inactivating and iC3b-degradation activity of native CFI.
  • the C3b-inactivating and iC3b-degradation activity of the fragment and/or variant of CFI and native CFI may be determined using any suitable method known to those of skill in the art. For example, using a proteolytic assay.
  • the CFI is a human CFI.
  • An example human CFI is the CFI having the UniProtKB accession number P05156.
  • the CFI may comprise or consist of the polypeptide sequence shown as SEQ ID NO: 13, or a variant which is at least 70% identical to SEQ ID NO: 13.
  • CFI polypeptide sequence (SEQ ID NO: 13): MKLLHVFLLFLCFHLRFCKVTYTSQEDLVEKKCLAKKYTHLSCDKVFCQPWQRCIEGTCVCKLPYQCP KNGTAVCATNRRSFPTYCQQKSLECLHPGTKFLNNGTCTAEGKFSVSLKHGNTDSEGIVEVKLVDQDK TMFICKSSWSMREANVACLDLGFQQGADTQRRFKLSDLSINSTECLHVHCRGLETSLAECTFTKRRTM GYQDFADVVCYTQKADSPMDDFFQCVNGKYISQMKACDGINDCGDQSDELCCKACQGKGFHCKSGVCI PSQYQCNGEVDCITGEDEVGCAGFASVTQEETEILTADMDAERRRIKSLLPKLSCGVKNRMHIRRKRI VGGKRAQLGDLPWQVAIKDASGITCGGIYIGGCWILTAAHCLRASKTHRYQIWTTVVDW
  • a transgene encoding CFI may comprise or consist of the polynucleotide sequence shown as SEQ ID NO: 14, or a variant which is at least 70% identical to SEQ ID NO: 14.
  • CFH Complement factor H
  • CFI complement factor B
  • C3b2Bb complement factor B
  • CFH not only inhibits formation of the convertases but it also shortens the lifespan of any convertase complex that forms.
  • CFH is a large (155 kDa) soluble glycoprotein.
  • CFH is composed from a total of 20 domains, each containing approximately 60 amino acid residues and termed complement control protein modules (CCPs) or short consensus repeats that are joined by short linkers consisting of 3–8 residues.
  • CCPs complement control protein modules
  • the CCP modules are numbered from 1–20 (from the N-terminus of the protein): CCPs 1–4 and CCPs 19–20 engage with C3b while CCPs 7 and CCPs 19–20 bind to GAGs and sialic acid.
  • the CFH or a fragment and/or variant thereof may be capable of binding C3b and/or C3d; and/or acting as a cofactor for the CFI-catalysed proteolytic cleavage of C3b; and/or increasing the irreversible dissociation of C3bBb and C3b2Bb into their separate components.
  • the fragment and/or variant of CFH may retain at least 50%, at least 60%, at least 70%, at least 80%, at least 90%, at least 95% or 100% of the activity of native CFH.
  • the activity of the fragment and/or variant of CFH and native CFH may be determined using any suitable method known to those of skill in the art.
  • the CFH is a human CFH.
  • CFH is the CFH having the UniProtKB accession number P08603.
  • the CFH may comprise or consist of the polypeptide sequence shown as SEQ ID NO: 15, or a variant which is at least 70% identical to SEQ ID NO: 15.
  • Illustrative CFH polypeptide sequence (SEQ ID NO: 15): MRLLAKIICLMLWAICVAEDCNELPPRRNTEILTGSWSDQTYPEGTQAIYKCRPGYRSLGNVIMVCRK GEWVALNPLRKCQKRPCGHPGDTPFGTFTLTGGNVFEYGVKAVYTCNEGYQLLGEINYRECDTDGWTN DIPICEVVKCLPVTAPENGKIVSSAMEPDREYHFGQAVRFVCNSGYKIEGDEEMHCSDDGFWSKEKPK CVEISCKSPDVINGSPISQKIIYKENERFQYKCNMGYEYSERGDAVCTESGWRPLPSCEEKSCDNPYI PNGDYSPLRIKHRTGDEITYQCRNGFYPATRGNTAKCTSTGWIPAPRCTLKPCDYPDIKHGGLYHENM RRPYFPVAVGKYYSYYCDEHFETPSGSYWDHIHCTQDGWSPAVPCLRKCYFPYLENGYNQNYGR
  • a transgene encoding CFH is NM_000186.4.
  • a transgene encoding CFH may comprise or consist of the polynucleotide sequence shown as SEQ ID NO: 16, or a variant which is at least 70% identical to SEQ ID NO: 16.
  • Illustrative CFH polynucleotide sequence (SEQ ID NO: 16): ATGAGACTTCTAGCAAAGATTATTTGCCTTATGTTATGGGCTATTTGTGTAGCAGAAGATTGCAATGA ACTTCCTCCAAGAAGAAATACAGAAATTCTGACAGGTTCCTGGTCTGACCAAACATATCCAGAAGGCA CCCAGGCTATCTATAAATGCCGCCCTGGATATAGATCTCTTGGAAATGTAATAATGGTATGCAGGAAG GGAGAATGGGTTGCTCTTAATCCATTAAGGAAATGTCAGAAAAGGCCCTGTGGACATCCTGGAGATAC TCCTTTTGGTACTTTTACCCTTACAGGAGGAAATGTGTTTGAATATGGTGTAAAAGCTGTGTATACAT GTAATGAGGGGTATCAATTGCTAGGTGAGATTAATTACCGTGAATGTGACACAGATGGATGGACCAAT GATATTCCTATATGAAGTTGTGAAGTGTTTACCAAT GATATTCCTATATGAAGT
  • the CFH fragment may be a splice variant.
  • complement factor H-like protein 1 FHL-1
  • FHL-1 complement factor H-like protein 1
  • CCPs 1-7 complement factor H-like protein 1-7
  • FHL-1 The transgene may encode FHL-1, or a fragment and/or variant thereof.
  • FHL-1 or a fragment and/or variant thereof may be capable of binding C3b and/or C3d.
  • the fragment and/or variant of FHL-1 may retain at least 50%, at least 60%, at least 70%, at least 80%, at least 90%, at least 95% or 100% of the activity of native FHL-1.
  • the activity of the fragment and/or variant of FHL-1 and native FHL-1 may be determined using any suitable method known to those of skill in the art.
  • the FHL-1 is a human FHL-1.
  • An example human FHL-1 is the FHL-1 having the NCBI Reference Sequence: NP_001014975.1.
  • the FHL-1 may comprise or consist of the polypeptide sequence shown as SEQ ID NO: 17, or a variant which is at least 70% identical to SEQ ID NO: 17.
  • FHL-1 polypeptide sequence (SEQ ID NO: 17): MRLLAKIICLMLWAICVAEDCNELPPRRNTEILTGSWSDQTYPEGTQAIYKCRPGYRSLGNVIMVCRK GEWVALNPLRKCQKRPCGHPGDTPFGTFTLTGGNVFEYGVKAVYTCNEGYQLLGEINYRECDTDGWTN DIPICEVVKCLPVTAPENGKIVSSAMEPDREYHFGQAVRFVCNSGYKIEGDEEMHCSDDGFWSKEKPK CVEISCKSPDVINGSPISQKIIYKENERFQYKCNMGYEYSERGDAVCTESGWRPLPSCEEKSCDNPYI PNGDYSPLRIKHRTGDEITYQCRNGFYPATRGNTAKCTSTGWIPAPRCTLKPCDYPDIKHGGLYHENM RRPYFPVAVGKYYSYYCDEHFETPSGSYWDHIHCTQDGWSPAVPCLRKCYFPYLENGYNQNHGRK
  • a transgene encoding FHL-1 is NM_001014975.2.
  • a transgene encoding FHL-1 may comprise or consist of the polynucleotide sequence shown as SEQ ID NO: 18, or a variant which is at least 70% identical to SEQ ID NO: 18.
  • FHL-1 polynucleotide sequence (SEQ ID NO: 18): ATGAGACTTCTAGCAAAGATTATTTGCCTTATGTTATGGGCTATTTGTGTAGCAGAAGATTGCAATGA ACTTCCTCCAAGAAGAAATACAGAAATTCTGACAGGTTCCTGGTCTGACCAAACATATCCAGAAGGCA CCCAGGCTATCTATAAATGCCGCCCTGGATATAGATCTCTTGGAAATGTAATAATGGTATGCAGGAAG GGAGAATGGGTTGCTCTTAATCCATTAAGGAAATGTCAGAAAAGGCCCTGTGGACATCCTGGAGATAC TCCTTTTGGTACTTTTACCCTTACAGGAGGAAATGTGTTTGAATATGGTGTAAAAGCTGTGTATACAT GTAATGAGGGGTATCAATTGCTAGGTGAGATTAATTACCGTGAATGTGACACAGATGGATGGACCAAT GATATTCCTATATGAAGTTGTGAAGTGTTTACCAAT GATATTCCTATATGAAGTTGTGAAGT
  • the polynucleotide or vector does not comprise a nephrotic syndrome- associated transgene, such as NPHS2, ADCK4, ALG1, ARHGAP24, ARGHDIA, CD151, CD2AP, COQ2, COQ6, DGKE, E2F3, EMP2, KANK2, LAGE3, LMNA, LMX1B, MAFB, NUP85, NUP93, NXF5, OSGEP, PAX2, PDSS2, PMM2, PODXL, SCARB2, SGPL1, Smad7, TP53RK, TPRKB, VDR, WDR73, WT1, ZMPSTE24, APOL1, NPHS1, TRPC6, NUP107, NUP133, NUP160, ACTN4, INF2, ANKFY1, ANLN, CRB2, ITGA3, KANK1, KANK4, MAGI2, MYO1E, OCRL, PTPRO, SMARCAL1, SYNPO, TBC1D8B, XPO5, T
  • the polynucleotide or vector does not comprise a vascular endothelial growth factor (VEGFC) transgene.
  • VEGFC vascular endothelial growth factor
  • the polynucleotide or vector does not comprise a COL4A3, COL4A4, or COL4A5 transgene.
  • the polynucleotide or vector does not comprise a nucleotide sequence encoding a complement protein, such as CFI, CFH, FHL-1, C1INH, C4BP, MASP2, C3, C5aR1, C5, C5a, CD55, CD35, CD46, CD59, vitronectin, or clusterin.
  • the polynucleotide or vector does not comprise a NPHS2, ADCK4, ALG1, ARHGAP24, ARGHDIA, CD151, CD2AP, COQ2, COQ6, DGKE, E2F3, EMP2, KANK2, LAGE3, LMNA, LMX1B, MAFB, NUP85, NUP93, NXF5, OSGEP, PAX2, PDSS2, PMM2, PODXL, SCARB2, SGPL1, Smad7, TP53RK, TPRKB, VDR, WDR73, WT1, ZMPSTE24, APOL1, NPHS1, TRPC6, NUP107, NUP133, NUP160, ACTN4, INF2, ANKFY1, ANLN, CRB2, ITGA3, KANK1, KANK4, MAGI2, MYO1E, OCRL, PTPRO, SMARCAL1, SYNPO, TBC1D8B, XPO5, TNS2.
  • the polynucleotide or vector may comprise one or more further regulatory sequences which may act pre- or post-transcriptionally.
  • the transgene may be operably linked to one or more further regulatory sequences.
  • the one or more further regulatory sequences may facilitate expression of the encoded polypeptide in glomerular cells (e.g. podocytes).
  • regulatory sequences are any sequences which facilitate expression of the polypeptides, e.g. act to increase expression of a transcript or to enhance mRNA stability. Suitable further regulatory sequences include for example enhancer elements, post-transcriptional regulatory elements and polyadenylation sites. Enhancers The polynucleotide or vector of the invention may comprise an enhancer. Suitably, the enhancer may be operably linked to the transgene. The enhancer may facilitate expression of the transgene in glomerular cells (e.g. podocytes). Suitably, the enhancer is a mammalian enhancer e.g. a human enhancer.
  • Enhancers are cis-acting. They can be located up to 1 Mbp (1,000,000 bp) away from the gene, upstream or downstream from the start site. Any suitable enhancer may be used, the selection of which may be readily made by the skilled person.
  • the polynucleotide or vector defined herein may comprise a tissue specific enhancer.
  • tissue-specific enhancer is an enhancer which preferentially facilitates expression of a gene in specific cells or tissues.
  • a tissue-specific enhancer may facilitate higher expression of a gene in specific cells-types as compared to other cell-types.
  • tissue-specific enhancer may be an enhancer which facilitates gene expression levels at least 10% higher, at least 20% higher, at least 30% higher, at least 40% higher, at least 50% higher, at least 100% higher, at least 200% higher, at least 300% higher, at least 400% higher, at least 500% higher, or at least 1000% higher in a specific cell-type compared to expression levels in other cell types.
  • Suitable tissue-specific enhancer will be well known to those of skill in the art.
  • the enhancer may be a kidney-specific enhancer, preferably a glomerular-specific enhancer, more preferably a podocyte-specific enhancer.
  • the enhancer may be operably linked to the transgene.
  • the enhancer may be or may be derived from an enhancer associated with a gene with selective expression in human kidney, glomerular cells, and/or podocytes. Methods to identify the enhancer regions associated with genes will be well known to those of skill in the art.
  • the podocyte-specific enhancer may be a NPHS1 or a NPHS2 enhancer, or a fragment or derivative thereof.
  • the podocyte-specific enhancer may be a NPHS1 enhancer, or a fragment or derivative thereof.
  • NPHS1 enhancer has been described in Guo, G., et al., 2004. Journal of the American Society of Nephrology, 15(11), pp.2851-2856.
  • a 186-bp fragment from the human NPHS1 promoter was capable of directing podocyte-specific expression of a ⁇ -galactosidase transgene when placed in front of a heterologous minimal promoter in transgenic mice.
  • the NPHS1 enhancer may comprise or consist of the nucleotide sequence shown as SEQ ID NO: 19, or a variant which is at least 70% identical to SEQ ID NO: 19.
  • NPHS1 enhancer (SEQ ID NO: 19): ctgctgagctgggagaccaccttgatctgacttctcccatcttcccagcctaagccaggccctggggt cacggaggctggggaggcaccgaggaacgcgcctggcatgtgctgacaggggattttatgctccagct gggccagctgggaggagcctgctgggcagaggccagagctgggggctctg
  • the variant may be at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 19.
  • the polynucleotide or vector may comprise a Kozak sequence.
  • the Kozak sequence may be operably linked to the transgene.
  • a Kozak sequence may be inserted before the start codon of the protein to improve the initiation of translation.
  • Suitable Kozak sequences will be well known to those of skill in the art.
  • the Kozak sequence may comprise or consist of the nucleotide sequence shown as SEQ ID NO: 20, or a variant which is at least 65% identical to SEQ ID NO: 20.
  • the variant may be at least 75%, at least 85%, or at least 90% identical to SEQ ID NO: 20.
  • the polynucleotide or vector defined herein may comprise a post-transcriptional regulatory element.
  • the post-transcriptional regulatory element may be operably linked to the transgene.
  • the post-transcriptional regulatory element may improve gene expression.
  • the polynucleotide may comprise a Woodchuck Hepatitis Virus Post-transcriptional Regulatory Element (WPRE).
  • WPRE Woodchuck Hepatitis Virus Post-transcriptional Regulatory Element
  • the WPRE may be operably linked to the transgene.
  • the WPRE sequence may have substitutions, deletions, or insertions within the X-antigen promoter and/or the initiation codon of the X-antigen. This may prevent the production of a functional X-antigen.
  • the WPRE may comprise or consist of the nucleotide sequence shown as SEQ ID NO: 21, or a variant which is at least 70% identical to SEQ ID NO: 21.
  • Exemplary WPRE (SEQ ID NO: 21): aatcaacctctggattacaaaatttgtgaaagattgactggtattcttaactatgttgctccttttac gctatgtggatacgctgctttaatgccttttgtatcatgctattgctttcccgtatggctttcattttct ccttgtataaatcctggttgctgtctctttatgaggagttgtggcccgttgtcaggcaacgtggcgtggc gtggc gtggcactgtgtttgctgacgcaacccccac
  • Polyadenylation signal The polynucleotide or vector defined herein may comprise a polyadenylation signal.
  • the polyadenylation signal may be operably linked to the transgene.
  • the polyadenylation signal may improve gene expression.
  • Suitable polyadenylation signals include the early SV40 polyadenylation signal (SV40pA), a chicken beta-globin polyadenylation signal, a bovine growth hormone polyadenylation signal (bGH), or a soluble neuropilin-1 polyadenylation signal.
  • the polyadenylation signal is an early SV40 polyadenylation signal (SV40pA) or a chicken beta- globin polyadenylation signal.
  • the polyadenylation signal is an early SV40 polyadenylation signal (SV40pA).
  • the polyadenylation signal may comprise or consist of the nucleotide sequence shown as SEQ ID NO: 22, or a variant which is at least 70% identical to SEQ ID NO: 22.
  • the variant may be at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99% identical to SEQ ID NO: 22.
  • Exemplary SV40pA signal sequence (SEQ ID NO: 22): aacttgtttattgcagcttataatggttacaaataaagcaatagcatcacaaatttcacaaataaagc attttttcactgcattctagttgtggtttgtccaaactcatcaatgtatcttatcatgtctggatc
  • the polyadenylation signal may comprise or consist of the nucleotide sequence shown as SEQ ID NO: 23, or a variant which is at least 70% identical to SEQ ID NO: 23.
  • the variant may be at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99% identical to SEQ ID NO: 23.
  • Exemplary bGH poly(A) signal sequence (SEQ ID NO: 23): ctgtgccttctagttgccagccatctgttgtttgcccctccccgtgccttccccgtgcctttccttgaccctggaaggt gccactcccactgtcctttcctaataaatgaggaaattgcatcgcattgtctgagtaggtgtctctgagtaggtgtgtcattc tattctggggggtggggggcaggacagcaagggggaggattgggaagacaatagcaggcatgct
  • the variant may be at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99% identical to SEQ ID NO: 24.
  • Exemplary soluble neuropilin-1 polyadenylation signal (SEQ ID NO: 24): aaataaaatacgaaatg
  • the polyadenylation signal may comprise or consist of the nucleotide sequence shown as SEQ ID NO: 25, or a variant which is at least 70% identical to SEQ ID NO: 25.
  • the variant may be at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99% identical to SEQ ID NO: 25.
  • Exemplary chicken beta-globin polyadenylation signal (SEQ ID NO: 25) Caataaaagatctttattttcattagatctgtgttggttttttgtgtgtgtgtg vectors
  • the present invention provides a vector comprising the promoter or polynucleotide described herein.
  • a vector is a tool that allows or facilitates the transfer of an entity from one environment to another.
  • the vector of the present invention is a viral vector.
  • the vector of the invention is preferably an adeno-associated viral (AAV) vector, although it is contemplated that other viral vectors may be used.
  • AAV adeno-associated viral
  • Other viral vectors include retroviral vectors, lentiviral vectors, adenoviral vectors, herpes simplex viral vectors, alphaviral vectors, flaviviral vectors, rhabdoviral vectors, measles viral vector, Newcastle disease viral vectors, poxviral vectors, and picornaviral vectors (see e.g. Lundstrom, K., 2018.
  • the vector of the present invention may be in the form of a viral vector particle.
  • the viral vector of the present invention is in the form of an AAV vector particle.
  • Methods of preparing and modifying viral vectors and viral vector particles, such as those derived from AAV, are well known in the art. Suitable methods are described in Ayuso, E., et al., 2010. Current gene therapy, 10(6), pp.423-436, Merten, O.W., et al., 2016. Molecular Therapy-Methods & Clinical Development, 3, p.16017; and Nadeau, I. and Kamen, A., 2003. Biotechnology advances, 20(7-8), pp.475-489.
  • the vector of the present invention may be capable of transducing kidney cells. In some embodiments, the vector of the present invention is capable of specifically transducing kidney cells.
  • the vector of the present invention is preferably capable of transducing glomerular cells (e.g. podocytes). In some embodiments, the vector of the present invention is capable of specifically transducing glomerular cells (e.g. podocytes).
  • the vector of the present invention is preferably capable of transducing podocytes. In some embodiments, the vector of the present invention is capable of specifically transducing podocytes.
  • Adeno-associated viral (AAV) vectors As described herein, the present invention provides an adeno-associated viral (AAV) comprising a transgene wherein the transgene is operably linked to a 5’-truncated podocin promoter.
  • AAV genome The AAV vector or AAV vector particle may comprise an AAV genome or a fragment or derivative thereof.
  • An AAV genome is a polynucleotide sequence, which may encode functions needed for production of an AAV particle. These functions include those operating in the replication and packaging cycle of AAV in a host cell, including encapsidation of the AAV genome into an AAV particle.
  • Naturally occurring AAVs are replication-deficient and rely on the provision of helper functions in trans for completion of a replication and packaging cycle.
  • the AAV genome of the AAV vector of the invention is typically replication-deficient.
  • the AAV genome may be in single-stranded form (ssAAV), either positive or negative-sense, or alternatively in double-stranded form (dsAAV).
  • ssAAV single-stranded form
  • dsAAV double-stranded form
  • the use of a double-stranded form allows bypass of the DNA replication step in the target cell and so can accelerate transgene expression.
  • the maximum packaging capacity of the single-stranded form is larger than the double-stranded form.
  • the AAV genome is in single-stranded form.
  • AAVs occurring in nature may be classified according to various biological systems.
  • the AAV genome may be from any naturally derived serotype, isolate or clade of AAV. AAV may be referred to in terms of their serotype.
  • a serotype corresponds to a variant subspecies of AAV which, owing to its profile of expression of capsid surface antigens, has a distinctive reactivity which can be used to distinguish it from other variant subspecies.
  • an AAV vector particle having a particular AAV serotype does not efficiently cross- react with neutralising antibodies specific for any other AAV serotype.
  • AAV serotypes include AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV10 and AAV11.
  • AAV may also be referred to in terms of clades or clones.
  • AAVs may be referred to in terms of a specific isolate, i.e. a genetic isolate of a specific AAV found in nature.
  • the term genetic isolate describes a population of AAVs which has undergone limited genetic mixing with other naturally occurring AAVs, thereby defining a recognisably distinct population at a genetic level.
  • the AAV genome of a naturally derived serotype, isolate or clade of AAV comprises at least one inverted terminal repeat sequence (ITR).
  • An ITR sequence acts in cis to provide a functional origin of replication and allows for integration and excision of the vector from the genome of a cell. ITRs may be the only sequences required in cis next to the therapeutic gene.
  • the AAV genome may also comprise packaging genes, such as rep and/or cap genes which encode packaging functions for an AAV particle.
  • a promoter may be operably linked to each of the packaging genes. Specific examples of such promoters include the p5, p19 and p40 promoters. For example, the p5 and p19 promoters are generally used to express the rep gene, while the p40 promoter is generally used to express the cap gene.
  • the rep gene encodes one or more of the proteins Rep78, Rep68, Rep52 and Rep40 or variants thereof.
  • the cap gene encodes one or more capsid proteins such as VP1, VP2 and VP3 or variants thereof. These proteins make up the capsid of an AAV particle, which determines the AAV serotype.
  • VP1, VP2, and VP3 may be produced by alternate mRNA splicing (Trempe, J.P. and Carter, B.J., 1988. Journal of virology, 62(9), pp.3356-3363).
  • VP1, VP2 and VP3 may have identical sequences, but wherein VP2 is truncated at the N-terminus relative to VP1, and VP3 is truncated at the N-terminus relative to VP2.
  • the AAV genome may be the full genome of a naturally occurring AAV.
  • a vector comprising a full AAV genome may be used to prepare an AAV vector or vector particle.
  • the AAV genome is derivatised for the purpose of administration to patients. Such derivatisation is standard in the art and the invention encompasses the use of any known derivative of an AAV genome, and derivatives which could be generated by applying techniques known in the art.
  • the AAV genome may be a derivative of any naturally occurring AAV.
  • the AAV genome is a derivative of AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV10, or AAV11.
  • the AAV genome is a derivative of AAV2.
  • Derivatives of an AAV genome include any truncated or modified forms of an AAV genome which allow for expression of a transgene from an AAV vector of the invention in vivo.
  • a derivative will include at least one inverted terminal repeat sequence (ITR), preferably more than one ITR, such as two ITRs or more.
  • ITR inverted terminal repeat sequence
  • One or more of the ITRs may be derived from AAV genomes having different serotypes, or may be a chimeric or mutant ITR.
  • a preferred mutant ITR is one having a deletion of a trs (terminal resolution site). This deletion allows for continued replication of the genome to generate a single-stranded genome which contains both coding and complementary sequences, i.e. a self-complementary AAV (scAAV) genome. This allows for bypass of DNA replication in the target cell, and so enables accelerated transgene expression. However, the maximum packaging capacity of a scAAV is reduced.
  • the AAV genome is not a scAAV genome.
  • the AAV genome may comprise one or more ITR sequences from any naturally derived serotype, isolate or clade of AAV or a variant thereof.
  • the AAV genome may comprise at least one, such as two, AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV10, or AAV11 ITRs, or variants thereof.
  • the AAV genome may comprise at least one, such as two, AAV2 ITRs.
  • the inclusion of one or more ITRs is preferred to aid concatamer formation of the AAV vector in the nucleus of a host cell, for example following the conversion of single-stranded vector DNA into double-stranded DNA by the action of host cell DNA polymerases.
  • ITR elements will be the only sequences retained from the native AAV genome in the derivative.
  • a derivative will preferably not include the rep and/or cap genes of the native genome and any other sequences of the native genome. This is preferred for the reasons described above, and also to reduce the possibility of integration of the vector into the host cell genome. Additionally, reducing the size of the AAV genome allows for increased flexibility in incorporating other sequence elements (such as regulatory elements) within the vector in addition to the transgene.
  • the invention also encompasses the replacement of one or more AAV sequences or genes with sequences from another virus or with chimeric genes composed of sequences from more than one virus.
  • Such chimeric genes may be composed of sequences from two or more related viral proteins of different viral species.
  • AAV serotype and capsid proteins The AAV vector particle may be encapsidated by capsid proteins.
  • the serotype may facilitate the transduction of glomerular cells (e.g. podocytes), for example, specific transduction of glomerular cells (e.g. podocytes).
  • the AAV vector particle may be a kidney-specific vector particle.
  • the AAV vector particle is a glomerular-specific (e.g. podocyte-specific) vector particle.
  • such ligands may include antibodies for targeting a particular cell surface receptor.
  • a derivative comprises capsid proteins i.e. VP1, VP2 and/or VP3
  • the derivative may be a chimeric, shuffled or capsid-modified derivative of one or more naturally occurring AAVs.
  • the invention encompasses the provision of capsid protein sequences from different serotypes, clades, clones, or isolates of AAV within the same vector (i.e. a pseudotyped vector).
  • the AAV vector may be in the form of a pseudotyped AAV vector particle. Chimeric, shuffled or capsid-modified derivatives will be typically selected to provide one or more desired functionalities for the AAV vector.
  • these derivatives may display increased efficiency of gene delivery, decreased immunogenicity (humoral or cellular), an altered tropism range and/or improved targeting of podocytes compared to an AAV vector comprising a naturally occurring AAV genome.
  • Increased efficiency of gene delivery may be effected by improved receptor or co-receptor binding at the cell surface, improved internalisation, improved trafficking within the cell and into the nucleus, improved uncoating of the viral particle and improved conversion of a single-stranded genome to double-stranded form.
  • Increased efficiency may also relate to an altered tropism range or targeting of podocytes, such that the vector dose is not diluted by administration to tissues where it is not needed.
  • a library of hybrid AAV genes created in this way by shuffling the capsid genes of several serotypes can be screened to identify viral clones having a desired functionality.
  • error prone PCR may be used to randomly mutate AAV capsid genes to create a diverse library of variants which may then be selected for a desired property.
  • the sequences of the capsid genes may also be genetically modified to introduce specific deletions, substitutions or insertions with respect to the native wild-type sequence.
  • capsid genes may be modified by the insertion of a sequence of an unrelated protein or peptide within an open reading frame of a capsid coding sequence, or at the N- and/or C-terminus of a capsid coding sequence.
  • the unrelated protein or peptide may advantageously be one which acts as a ligand for a particular cell type, thereby conferring improved binding to a target cell or improving the specificity of targeting of the vector to a particular cell population.
  • the unrelated protein may also be one which assists purification of the viral particle as part of the production process, i.e. an epitope or affinity tag.
  • the site of insertion will typically be selected so as not to interfere with other functions of the viral particle e.g. internalisation, trafficking of the viral particle.
  • the capsid protein may be an artificial or mutant capsid protein.
  • artificial capsid means that the capsid particle comprises an amino acid sequence which does not occur in nature or which comprises an amino acid sequence which has been engineered (e.g. modified) from a naturally occurring capsid amino acid sequence.
  • the artificial capsid protein comprises a mutation or a variation in the amino acid sequence compared to the sequence of the parent capsid from which it is derived where the artificial capsid amino acid sequence and the parent capsid amino acid sequences are aligned.
  • the capsid protein may comprise a mutation or modification relative to the wild type capsid protein which improves the ability to transduce podocytes relative to an unmodified or wild type viral particle.
  • Improved ability to transduce podocytes may be measured for example by measuring the expression of a transgene, e.g. GFP, carried by the AAV vector particle, wherein expression of the transgene in podocytes correlates with the ability of the AAV vector particle to transduce podocytes.
  • the AAV vector particle may be an AAV3B, LK03, AAV9, ShH10, AAV-DJ, AAV2, AAV6.2, AAV5, or AAV8 vector particle.
  • the AAV vector particle is an AAV3B vector particle or an LK03 vector particle.
  • the AAV vector particle is an LK03 vector particle.
  • the AAV vector particle is a ShH10, AAV-DJ, AAV2, AAV6.2, or AAV5 vector particle.
  • the AAV vector particle may comprise an AAV3B, LK03, AAV9, ShH10, AAV-DJ, AAV2, AAV6.2, AAV5, or AAV8 capsid protein.
  • the AAV vector particle comprises an AAV3B capsid protein or an LK03 capsid protein.
  • the AAV vector particle comprises an LK03 capsid protein.
  • the AAV vector particle comprises a ShH10, AAV-DJ, AAV2, AAV6.2, or AAV5 capsid protein.
  • the AAV vector particle may comprise AAV3B, LK03, AAV9, ShH10, AAV-DJ, AAV2, AAV6.2, AAV5, or AAV8 capsid proteins VP1, VP2 and VP3.
  • the AAV vector particle comprises AAV3B or LK03 capsid proteins VP1, VP2 and VP3.
  • the AAV vector particle comprises LK03 capsid proteins VP1, VP2 and VP3.
  • the AAV vector particle comprises ShH10, AAV-DJ, AAV2, AAV6.2, or AAV5 capsid proteins VP1, VP2 and VP3.
  • the AAV vector particle may have an AAV2 genome and AAV3B capsid proteins (AAV2/3B), an AAV2 genome and LK03 capsid proteins, an AAV2 genome and AAV9 capsid proteins (AAV2/9), an AAV2 genome and ShH10 capsid proteins, an AAV2 genome and AAV-DJ capsid proteins, an AAV2 genome and AAV2 capsid proteins, an AAV2 genome and AAV6.2 capsid proteins, an AAV2 genome and AAV5 capsid proteins, or an AAV2 genome and AAV8 capsid proteins (AAV2/8).
  • AAV2/3B AAV2 genome and AAV3B capsid proteins
  • AAV2 genome and LK03 capsid proteins an AAV2 genome and AAV9 capsid proteins (AAV2/9)
  • AAV2 genome and ShH10 capsid proteins an AAV2 genome and AAV-DJ capsid proteins
  • an AAV2 genome and AAV2 capsid proteins an AAV2 genome
  • AAVX/Y may denote a pseudotyped AAV, for example where the ITR sequences are from AAVX and flank a cassette harbouring a payload which is encapsidated into serotype AAVY (i.e. with AAVY capsid proteins).
  • AAV3B serotype The AAV vector particle may comprise an AAV3B capsid protein.
  • the AAV vector particle may be encapsidated by AAV3B capsid proteins.
  • Two distinct AAV3 isolates (AAV3A and AAV3B) have been cloned. In comparison with vectors based on other AAV serotypes, it is thought that AAV3 vectors inefficiently transduce most cell types.
  • AAV3B may efficiently transduce podocytes.
  • AA3B has been described in Rutledge, E.A., et al., 1998. Journal of virology, 72(1), pp.309-319.
  • the AAV vector particle may comprise an AAV3B VP1 capsid protein, an AAV3B VP2 capsid protein, and/or an AAV3B VP3 capsid protein.
  • the AAV vector particle may be encapsidated by AAV3B VP1 capsid proteins, AAV3B VP2 capsid proteins, and/or AAV3B VP3 capsid proteins.
  • the AAV vector particle may be encapsidated by AAV3B VP1, VP2, and VP3 capsid proteins.
  • the AAV3B VP1 capsid protein may comprise or consist of the amino acid sequence shown as SEQ ID NO: 26, or a variant which is at least 90% identical to SEQ ID NO: 26.
  • Exemplary AAV3B VP1 capsid protein (SEQ ID NO: 26): MAADGYLPDWLEDNLSEGIREWWALKPGVPQPKANQQHQDNRRGLVLPGYKYLGPGNGLDKGEPVNEA DAAALEHDKAYDQQLKAGDNPYLKYNHADAEFQERLQEDTSFGGNLGRAVFQAKKRILEPLGLVEEAA KTAPGKKRPVDQSPQEPDSSSGVGKSGKQPARKRLNFGQTGDSESVPDPQPLGEPPAAPTSLGSNTMA SGGGAPMADNNEGADGVGNSSGNWHCDSQWLGDRVITTSTRTWALPTYNNHLYKQISSQSGASNDNHY FGYSTPWGYFDFNRFHCHFSPRDWQRLINNNWGFRPKKLSFKLFNIQVKEVTQNDGTTTIANNLTSTV QVFTDSEYQLPYVLGSAHQGCLPPFPADVFMVPQYGYLTLNNGSQAVGRSSFYCLEY
  • the AAV3B VP2 and VP3 capsid proteins may be N-terminal truncations of SEQ ID NO: 26, or N-terminal truncations of a variant which is at least 90% identical, at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 26.
  • LK03 serotype The AAV vector particle may comprise an LK03 capsid protein.
  • the AAV vector particle may be encapsidated by LK03 capsid proteins.
  • the AAV-LK03 cap sequence consists of fragments from seven different wild-type serotypes (AAV1, 2, 3B, 4, 6, 8, 9) and is described in Lisowski, L., et al., 2014. Nature, 506(7488), pp.382-386.
  • the present inventors have demonstrated that AAV-LK03 vectors can achieve high transduction of close to 100% in human podocytes in vitro.
  • the AAV vector particle may comprise an LK03 VP1 capsid protein, an LK03 VP2 capsid protein, and/or an LK03 VP3 capsid protein.
  • the AAV vector particle may be encapsidated by LK03 VP1 capsid proteins, LK03 VP2 capsid proteins, and/or LK03 VP3 capsid proteins.
  • the AAV vector particle may be encapsidated by LK03 VP1, VP2, and VP3 capsid proteins.
  • the LK03 VP1 capsid protein may comprise or consist of the amino acid sequence shown as SEQ ID NO: 27, or a variant which is at least 90% identical to SEQ ID NO: 27.
  • Exemplary LK03 VP1 capsid protein (SEQ ID NO: 27): MAADGYLPDWLEDNLSEGIREWWALQPGAPKPKANQQHQDNARGLVLPGYKYLGPGNGLDKGEPVNAA DAAALEHDKAYDQQLKAGDNPYLKYNHADAEFQERLKEDTSFGGNLGRAVFQAKKRLLEPLGLVEEAA KTAPGKKRPVDQSPQEPDSSSGVGKSGKQPARKRLNFGQTGDSESVPDPQPLGEPPAAPTSLGSNTMA SGGGAPMADNNEGADGVGNSSGNWHCDSQWLGDRVITTSTRTWALPTYNNHLYKQISSQSGASNDNHY FGYSTPWGYFDFNRFHCHFSPRDWQRLINNNWGFRPKKLSFKLFNIQVKEVTQNDGTTTIANNLTSTV QVFTDSEYQLPYVLGSAHQGCLPPFPADVFMVPQYGYLTLNNGSQAVGRSSFYCLEYFPSQ
  • the LK03 VP2 and VP3 capsid proteins may be N-terminal truncations of SEQ ID NO: 27, or N-terminal truncations of a variant which is at least 90% identical, at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 27.
  • AAV9 serotype The AAV vector particle may comprise an AAV9 capsid protein.
  • the AAV vector particle may be encapsidated by AAV9 capsid proteins. The present inventors have demonstrated that AAV9 vectors can achieve high transduction in human podocytes in vivo.
  • Exemplary AAV9 VP1 capsid protein (SEQ ID NO: 28): MAADGYLPDWLEDNLSEGIREWWALKPGAPQPKANQQHQDNARGLVLPGYKYLGPGNGLDKGEPVNAA DAAALEHDKAYDQQLKAGDNPYLKYNHADAEFQERLKEDTSFGGNLGRAVFQAKKRLLEPLGLVEEAA KTAPGKKRPVEQSPQEPDSSAGIGKSGAQPAKKRLNFGQTGDTESVPDPQPIGEPPAAPSGVGSLTMA SGGGAPVADNNEGADGVGSSSGNWHCDSQWLGDRVITTSTRTWALPTYNNHLYKQISNSTSGGSSNDN AYFGYSTPWGYFDFNRFHCHFSPRDWQRLINNNWGFRPKRLNFKLFNIQVKEVTDNNGVKTIANNLTS TVQVFTDSDYQLPYVLGSAHEGCLPPFPADVFMIPQYGYLTLNDGSQAVGRSSFYCLEYFPSQMLRT
  • the AAV9 VP2 and VP3 capsid proteins may be N-terminal truncations of SEQ ID NO: 28, or N-terminal truncations of a variant which is at least 90% identical, at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 28.
  • ShH10 serotype The AAV vector particle may comprise a ShH10 capsid protein.
  • the AAV vector particle may be encapsidated by ShH10 capsid proteins.
  • the ShH10 variant is derived from AAV6 and has increased specificity and efficiency for Müller cells (see e.g. Klimczak, R.R., et al., 2009.
  • the AAV vector particle may comprise a ShH10 VP1 capsid protein, a ShH10 VP2 capsid protein, and/or a ShH10 VP3 capsid protein.
  • the AAV vector particle may be encapsidated by ShH10 VP1 capsid proteins, ShH10 VP2 capsid proteins, and/or ShH10 VP3 capsid proteins.
  • the AAV vector particle may be encapsidated by ShH10 VP1, VP2, and VP3 capsid proteins.
  • the ShH10 VP1 capsid protein may comprise or consist of the amino acid sequence shown as SEQ ID NO: 29, or a variant which is at least 90% identical to SEQ ID NO: 29.
  • Exemplary ShH10 VP1 capsid protein (SEQ ID NO: 29): MAADGYLPDWLEDNLSEGIREWWDLKPGAPKPKANQQKQDDGRGLVLPGYKYLGPFNGLDKGEPVNAA DAAALEHDKAYDQQLKAGDNPYLRYNHADAEFQERLQEDTSFGGNLGRAVFQAKKRVLEPFGLVEEGA KTAPGKKRPVEQSPQEPDSSSGIGKTGQQPAKKRLNFGQTGDSESVPDPQPLGEPPATPAAVGPTTMA SGGGAPMADNNEGADGVGNASGNWHCDSTWLGDRVITTSTRTWALPTYNNHLYKQISSASTGASNDNH YFGYSTPWGYFDFNRFHCHFSPRDWQRLINNNWGFRPKRLNFKLFNVQ
  • the ShH10 VP2 and VP3 capsid proteins may be N-terminal truncations of SEQ ID NO: 29, or N-terminal truncations of a variant which is at least 90% identical, at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 29.
  • AAV-DJ serotype The AAV vector particle may comprise an AAV-DJ capsid protein.
  • the AAV vector particle may be encapsidated by AAV-DJ capsid proteins.
  • AAV-DJ is a hybrid vector created from DNA shuffling of eight AAV serotypes, which mediates efficient gene expression both in vitro and in vivo (see e.g.
  • the AAV vector particle may comprise an AAV-DJ VP1 capsid protein, an AAV-DJ VP2 capsid protein, and/or an AAV-DJ VP3 capsid protein.
  • the AAV vector particle may be encapsidated by AAV-DJ VP1 capsid proteins, AAV-DJ VP2 capsid proteins, and/or AAV-DJ VP3 capsid proteins.
  • the AAV vector particle may be encapsidated by AAV-DJ VP1, VP2, and VP3 capsid proteins.
  • the AAV-DJ VP1 capsid protein may comprise or consist of the amino acid sequence shown as SEQ ID NO: 30, or a variant which is at least 90% identical to SEQ ID NO: 30.
  • Exemplary AAV-DJ VP1 capsid protein (SEQ ID NO: 30): MAADGYLPDWLEDTLSEGIRQWWKLKPGPPPPKPAERHKDDSRGLVLPGYKYLGPFNGLDKGEPVNEA DAAALEHDKAYDRQLDSGDNPYLKYNHADAEFQERLKEDTSFGGNLGRAVFQAKKRLLEPLGLVEEAA KTAPGKKRPVEHSPVEPDSSSGTGKAGQQPARKRLNFGQTGDADSVPDPQPIGEPPAAPSGVGSLTMA AGGGAPMADNNEGADGVGNSSGNWHCDSTWMGDRVITTSTRTWALPTYNNHLYKQISNSTSGGSSNDN AYFGYSTPWGYFDFNRFHCHFSPRDWQRLINNNWGFRPKRLSFKLF
  • the AAV-DJ VP2 and VP3 capsid proteins may be N-terminal truncations of SEQ ID NO: 30, or N-terminal truncations of a variant which is at least 90% identical, at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 30.
  • AAV2 serotype The AAV vector particle may comprise an AAV2 capsid protein.
  • the AAV vector particle may be encapsidated by AAV2 capsid proteins.
  • the AAV vector particle may comprise an AAV2 VP1 capsid protein, an AAV2 VP2 capsid protein, and/or an AAV2 VP3 capsid protein.
  • the AAV vector particle may be encapsidated by AAV2 VP1 capsid proteins, AAV2 VP2 capsid proteins, and/or AAV2 VP3 capsid proteins.
  • the AAV vector particle may be encapsidated by AAV2 VP1, VP2, and VP3 capsid proteins.
  • the AAV2 VP1 capsid protein may comprise or consist of the amino acid sequence shown as SEQ ID NO: 31, or a variant which is at least 90% identical to SEQ ID NO: 31.
  • Exemplary AAV2 VP1 capsid protein (SEQ ID NO: 31): MAADGYLPDWLEDTLSEGIRQWWKLKPGPPPPKPAERHKDDSRGLVLPGYKYLGPFNGLDKGEPVNEA DAAALEHDKAYDRQLDSGDNPYLKYNHADAEFQERLKEDTSFGGNLGRAVFQAKKRVLEPLGLVEEPV KTAPGKKRPVEHSPVEPDSSSGTGKAGQQPARKRLNFGQTGDADSVPDPQPLGQPPAAPSGLGTNTMA TGSGAPMADNNEGADGVGNSSGNWHCDSTWMGDRVITTSTRTWALPTYNNHLYKQISSQSGASNDNHY FGYSTPWGYFDFNRFHCHFSPRDWQRLINNNWGFRPKRLNFKLFNIQVKEVTQNDGTTTIANNLTSTV QVFTDSEYQLPYVLGSAHQGCLPPFPADVFMVPQYGYLTLNNGSQAVGRSSFYCLEYFPSQMLRT
  • the AAV2 VP2 and VP3 capsid proteins may be N-terminal truncations of SEQ ID NO: 31, or N-terminal truncations of a variant which is at least 90% identical, at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 31.
  • AAV6.2 serotype The AAV vector particle may comprise an AAV6.2 capsid protein.
  • the AAV vector particle may be encapsidated by AAV6.2 capsid proteins.
  • the AAV6.2 vector mutant was created by mutating the phenyalanine (F) residue at position 129 in AAV6 to leucine (L) (see e.g.
  • the AAV vector particle may comprise an AAV6.2 VP1 capsid protein, an AAV6.2 VP2 capsid protein, and/or an AAV6.2 VP3 capsid protein.
  • the AAV vector particle may be encapsidated by AAV6.2 VP1 capsid proteins, AAV6.2 VP2 capsid proteins, and/or AAV6.2 VP3 capsid proteins.
  • the AAV vector particle may be encapsidated by AAV6.2 VP1, VP2, and VP3 capsid proteins.
  • the AAV6.2 VP1 capsid protein may comprise or consist of the amino acid sequence shown as SEQ ID NO: 32, or a variant which is at least 90% identical to SEQ ID NO: 32.
  • Exemplary AAV6.2 VP1 capsid protein (SEQ ID NO: 32): MAADGYLPDWLEDNLSEGIREWWDLKPGAPKPKANQQKQDDGRGLVLPGYKYLGPFNGLDKGEPVNAA DAAALEHDKAYDQQLKAGDNPYLRYNHADAEFQERLQEDTSFGGNLGRAVFQAKKRVLEPLGLVEEGA KTAPGKKRPVEQSPQEPDSSSGIGKTGQQPAKKRLNFGQTGDSESVPDPQPLGEPPATPAAVGPTTMA SGGGAPMADNNEGADGVGNASGNWHCDSTWLGDRVITTSTRTWALPTYNNHLYKQISSASTGASNDNH YFGYSTPWGYFDFNRFHCHFSPRDWQRLINNNWGFRPKRLNFKLFNI
  • the AAV6.2 VP2 and VP3 capsid proteins may be N-terminal truncations of SEQ ID NO: 32, or N-terminal truncations of a variant which is at least 90% identical, at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 32.
  • AAV5 serotype The AAV vector particle may comprise an AAV5 capsid protein.
  • the AAV vector particle may be encapsidated by AAV5 capsid proteins.
  • the AAV vector particle may comprise an AAV5 VP1 capsid protein, an AAV5 VP2 capsid protein, and/or an AAV5 VP3 capsid protein.
  • the AAV vector particle may be encapsidated by AAV5 VP1 capsid proteins, AAV5 VP2 capsid proteins, and/or AAV5 VP3 capsid proteins.
  • the AAV vector particle may be encapsidated by AAV5 VP1, VP2, and VP3 capsid proteins.
  • the AAV5 VP1 capsid protein may comprise or consist of the amino acid sequence shown as SEQ ID NO: 33, or a variant which is at least 90% identical to SEQ ID NO: 33.
  • Exemplary AAV5 VP1 capsid protein (SEQ ID NO: 33): MSFVDHPPDWLEEVGEGLREFLGLEAGPPKPKPNQQHQDQARGLVLPGYNYLGPGNGLDRGEPVNRAD EVAREHDISYNEQLEAGDNPYLKYNHADAEFQEKLADDTSFGGNLGKAVFQAKKRVLEPFGLVEEGAK TAPTGKRIDDHFPKRKKARTEEDSKPSTSSDAEAGPSGSQQLQIPAQPASSLGADTMSAGGGGPLGDN NQGADGVGNASGDWHCDSTWMGDRVVTKSTRTWVLPSYNNHQYREIKSGSVDGSNANAYFGYSTPWGY FDFNRFHSHWSPRDWQRLINNYWGFRPRSLRVKIFNIQVKEVTVQDSTTTIANNLTSTVQVFTDDDYQ LPYVVGNGTEGCLPAFPPQVFTLPQYGYATLNRDNTENPTERSSFFCLEYFPSKMLRTGNNFEFTY
  • the AAV5 VP2 and VP3 capsid proteins may be N-terminal truncations of SEQ ID NO: 33, or N-terminal truncations of a variant which is at least 90% identical, at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 33.
  • Variants, derivatives, analogues, homologues and fragments In addition to the specific proteins (e.g., encoded by the transgenes) and nucleotides (e.g., promoters) mentioned herein, the invention also encompasses variants, derivatives, homologues and fragments thereof.
  • a “variant” of any given sequence is a sequence in which the specific sequence of residues (whether amino acid or nucleic acid residues) has been modified in such a manner that the polypeptide or polynucleotide in question retains at least one of its endogenous functions.
  • a variant sequence can be obtained by addition, deletion, substitution, modification, replacement and/or variation of at least one residue present in the naturally occurring polypeptide or polynucleotide.
  • a variant promoter sequence retains at least some level of the activity and specificity of the promoter sequence from which it is obtained.
  • derivative as used herein in relation to proteins or polypeptides of the invention includes any substitution of, variation of, modification of, replacement of, deletion of and/or addition of one (or more) amino acid residues from or to the sequence, providing that the resultant protein or polypeptide retains at least one of its endogenous functions.
  • amino acid substitutions may be made, for example from 1, 2 or 3, to 10 or 20 substitutions, provided that the modified sequence retains the required activity or ability.
  • Amino acid substitutions may include the use of non-naturally occurring analogues.
  • nucleic acid substitutions may be made, for example from 1, 2 or 3, to 10 or 20 substitutions (e.g.1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, or 20 substitutions), provided that the modified sequence retains the required activity or ability.
  • Proteins used in the invention may also have deletions, insertions or substitutions of amino acid residues which produce a silent change and result in a functionally equivalent protein. Deliberate amino acid substitutions may be made on the basis of similarity in polarity, charge, solubility, hydrophobicity, hydrophilicity and/or the amphipathic nature of the residues as long as the endogenous function is retained.
  • negatively charged amino acids include aspartic acid and glutamic acid; positively charged amino acids include lysine and arginine; and amino acids with uncharged polar head groups having similar hydrophilicity values include asparagine, glutamine, serine, threonine and tyrosine.
  • Conservative substitutions may be made, for example according to the table below.
  • Amino acids in the same block in the second column and preferably in the same line in the third column may be substituted for each other:
  • the term “homologue” as used herein means a variant having a certain homology with the wild type amino acid sequence or the wild type nucleotide sequence.
  • the term “homology” can be equated with “identity”.
  • a homologous sequence is taken to include an amino acid sequence which may be at least 50%, 55%, 65%, 75%, 85% or 90% identical, preferably at least 95%, 96% or 97% or 98% or 99% identical to the subject sequence.
  • the homologues will comprise the same active sites etc. as the subject amino acid sequence.
  • homology can also be considered in terms of similarity (i.e. amino acid residues having similar chemical properties/functions), in the context of the present invention it is preferred to express homology in terms of sequence identity.
  • a homologous sequence is taken to include a nucleotide sequence which may be at least 50%, 55%, 65%, 75%, 85% or 90% identical, preferably at least 95%, 96% or 97% or 98% or 99% identical to the subject sequence.
  • homology can also be considered in terms of similarity, in the context of the present invention it is preferred to express homology in terms of sequence identity.
  • reference to a sequence which has a percent identity to any one of the SEQ ID NOs detailed herein refers to a sequence which has the stated percent identity over the entire length of the SEQ ID NO referred to. Homology comparisons can be conducted by eye, or more usually, with the aid of readily available sequence comparison programs.
  • Percent homology may be calculated over contiguous sequences, i.e. one sequence is aligned with the other sequence and each amino acid or nucleotide in one sequence is directly compared with the corresponding amino acid or nucleotide in the other sequence, one residue at a time. This is called an “ungapped” alignment. Typically, such ungapped alignments are performed only over a relatively short number of residues.
  • the default gap penalty for amino acid sequences is -12 for a gap and -4 for each extension. Calculation of maximum percent homology therefore firstly requires the production of an optimal alignment, taking into consideration gap penalties.
  • a suitable computer program for carrying out such an alignment is the GCG Wisconsin Bestfit package (University of Wisconsin, USA; Devereux et al. (1984) Nucleic Acids Research 12: 387). Examples of other software that can perform sequence comparisons include, but are not limited to, the BLAST package (see Ausubel et al. (1999) ibid – Ch.18), FASTA (Atschul et al. (1990) J. Mol. Biol.
  • BLAST and FASTA are available for offline and online searching (see Ausubel et al. (1999) ibid, pages 7-58 to 7- 60). However, for some applications, it is preferred to use the GCG Bestfit program.
  • Another tool, BLAST 2 Sequences is also available for comparing protein and nucleotide sequences (FEMS Microbiol. Lett. (1999) 174(2):247-50; FEMS Microbiol. Lett. (1999) 177(1):187-8).
  • the alignment process itself is typically not based on an all-or-nothing pair comparison. Instead, a scaled similarity score matrix is generally used that assigns scores to each pairwise comparison based on chemical similarity or evolutionary distance.
  • An example of such a matrix commonly used is the BLOSUM62 matrix (the default matrix for the BLAST suite of programs).
  • GCG Wisconsin programs generally use either the public default values or a custom symbol comparison table if supplied (see the user manual for further details). For some applications, it is preferred to use the public default values for the GCG package, or in the case of other software, the default matrix, such as BLOSUM62.
  • “Fragments” are also variants and the term typically refers to a selected region of the polypeptide or polynucleotide that is of interest either functionally or, for example, in an assay. “Fragment” thus refers to an amino acid or nucleic acid sequence that is a portion of a full- length polypeptide or polynucleotide. Such variants, derivatives, homologues and fragments may be prepared using standard recombinant DNA techniques such as site-directed mutagenesis.
  • the present invention provides a cell comprising the promoter, polynucleotide, or vector described herein.
  • the cell may be an isolated cell.
  • the cell may be a human cell, suitably an isolated human cell.
  • the cell may be any cell type known in the prior art.
  • the cell may be a kidney cell or glomerular cell, for example a podocyte.
  • the cell may be an immortalized kidney cell or glomerular cell, for example an immortalized podocyte.
  • Suitable podocyte cell lines will be well known to those of skill in the art, for example CIHP-1. Methods to generate immortalized podocytes will be well known to those of skill in the art. Suitable methods are described in Ni, L., et al., 2012. Nephrology, 17(6), pp.525-531.
  • the cell may be a producer cell.
  • the term “producer cell” includes a cell that produces viral particles, after transient transfection, stable transfection or vector transduction of all the elements necessary to produce the viral particles or any cell engineered to stably comprise the elements necessary to produce the viral particles.
  • Suitable producer cells will be well known to those of skill in the art. Suitable producer cell lines include HEK 293 (e.g. HEK 293T), HeLa, and A549 cell lines.
  • the cell may be a packaging cell.
  • the term “packaging cell” includes a cell which contains some or all of the elements necessary for packaging an infectious recombinant virus.
  • the packaging cell may lack a recombinant viral vector genome. Typically, such packaging cells contain one or more vectors which are capable of expressing viral structural proteins.
  • the present invention provides a pharmaceutical composition comprising the polynucleotide, vector, or cell defined herein.
  • the vector is an AAV vector particle.
  • a pharmaceutical composition is a composition that comprises or consists of a therapeutically effective amount of a pharmaceutically active agent i.e. the AAV vector.
  • a pharmaceutically acceptable carrier preferably includes a pharmaceutically acceptable carrier, diluent or excipient (including combinations thereof).
  • pharmaceutically acceptable is included that the formulation is sterile and pyrogen free.
  • the carrier, diluent, and/or excipient must be “acceptable” in the sense of being compatible with the vector and not deleterious to the recipients thereof.
  • the carriers, diluents, and excipients will be saline or infusion media which will be sterile and pyrogen free; however, other acceptable carriers, diluents, and excipients may be used. Acceptable carriers, diluents, and excipients for therapeutic use are well known in the pharmaceutical art.
  • compositions may comprise as - or in addition to - the carrier, excipient or diluent any suitable binder(s), lubricant(s), suspending agent(s), coating agent(s) or solubilising agent(s).
  • Examples of pharmaceutically acceptable carriers include, for example, water, salt solutions, alcohol, silicone, waxes, petroleum jelly, vegetable oils, polyethylene glycols, propylene glycol, liposomes, sugars, gelatin, lactose, amylose, magnesium stearate, talc, surfactants, silicic acid, viscous paraffin, perfume oil, fatty acid monoglycerides and diglycerides, petroethral fatty acid esters, hydroxymethyl-cellulose, polyvinylpyrrolidone, and the like.
  • the vector, cell, or pharmaceutical composition according to the present invention may be administered in a manner appropriate for treating and/or preventing the diseases described herein.
  • the quantity and frequency of administration will be determined by such factors as the condition of the subject, and the type and severity of the subject's disease, although appropriate dosages may be determined by clinical trials.
  • the pharmaceutical composition may be formulated accordingly.
  • the vector, cell or pharmaceutical composition according to the present invention may be administered parenterally, for example, intravenously, or by infusion techniques.
  • the vector, cell or pharmaceutical composition may be administered in the form of a sterile aqueous solution which may contain other substances, for example, enough salts or glucose to make the solution isotonic with blood.
  • the aqueous solution may be suitably buffered (preferably to a pH of from 3 to 9).
  • the pharmaceutical composition may be formulated accordingly.
  • the preparation of suitable parenteral formulations under sterile conditions is readily accomplished by standard pharmaceutical techniques well-known to those skilled in the art.
  • the vector, cell or pharmaceutical composition according to the present invention may be administered systemically, for example by intravenous injection.
  • the vector, cell or pharmaceutical composition according to the present invention may be administered locally, for example by targeting administration to the kidney.
  • the vector, cell or pharmaceutical composition may be administered by injection into the renal artery or by ureteral or subcapsular injection.
  • the pharmaceutical compositions may comprise vectors or cells of the invention in infusion media, for example sterile isotonic solution.
  • the pharmaceutical composition may be enclosed in ampoules, disposable syringes or multiple dose vials made of glass or plastic.
  • the vector, cell or pharmaceutical composition may be administered in a single or in multiple doses. Particularly, the vector, cell or pharmaceutical composition may be administered in a single, one-off dose.
  • the pharmaceutical composition may be formulated accordingly.
  • the vector, cell or pharmaceutical composition may be administered at varying doses (e.g. measured in vector genomes (vg) per kg). The physician in any event will determine the actual dosage which will be most suitable for any individual subject and it will vary with the age, weight and response of the particular subject.
  • kits comprising the vector, cells and/or pharmaceutical composition of the present invention.
  • said kits are for use in the methods and used as described herein, e.g., the therapeutic methods as described herein.
  • said kits comprise instructions for use of the kit components.
  • the present invention provides the vector, cell or pharmaceutical composition described herein for use as a medicament.
  • the present invention provides use of the vector, cell or pharmaceutical composition described herein in the manufacture of a medicament.
  • the present invention provides a method of administering the vector, cell or pharmaceutical composition described herein to a subject in need thereof.
  • Glomerular diseases The vector, cell or pharmaceutical composition described herein may be used to treat glomerular diseases in a subject.
  • the subject is a human subject.
  • the present invention provides the vector, cell or pharmaceutical composition described herein for use in preventing or treating a kidney disease, for example a glomerular disease.
  • the present invention provides use of the vector, cell or pharmaceutical composition described herein for the manufacture of a medicament for preventing or treating a glomerular disease.
  • the present invention provides a method of preventing or treating a glomerular disease comprising administering the vector, cell or pharmaceutical composition described herein to a subject in need thereof.
  • Glomerular diseases can be classified as either nephrotic or nephritic. Nephrotic syndrome often involves factors that affect the integrity of podocyte–podocyte or podocyte–GBM interactions.
  • the glomerular disease is a genetic glomerular disease, i.e. a glomerular disease which is inherited.
  • Genetic glomerular diseases include podocyte-associated genetic glomerular diseases, such as nephrotic syndrome, and GBM-associated glomerular diseases, such as Alport Syndrome.
  • the glomerular disease is a podocyte-associated genetic glomerular disease.
  • Podocyte-associated genetic glomerular diseases include Congenital nephrotic syndrome of the Finnish type, Congenital nephrotic syndrome type 2, Familial nephrotic syndrome type 3, Frasier syndrome and Denys–Drash syndrome, Schimke immuno-osseous dysplasia, Nephrotic syndrome caused by mutations in CD2AP, Nephrotic syndrome caused by mutations in actinin-4, Nephrotic syndrome caused by mutations in TRPC6, and Epstein and Fechtner syndrome.
  • the glomerular disease is nephrotic syndrome.
  • the glomerular disease is a GBM-associated genetic glomerular disease.
  • Podocyte-associated genetic glomerular diseases include X-linked Alport syndrome, Autosomal recessive Alport syndrome, Autosomal dominant Alport syndrome, Thin basement membrane diseases, Pierson syndrome, and Nail–patella syndrome.
  • the glomerular disease is Alport syndrome (AS).
  • AS is also known as familial nephritis, hereditary nephritis, thin basement membrane disease and thin basement membrane nephropathy.
  • Example 1 Transfection of HEK293T cells
  • the following constructs were tested: ⁇ A construct comprising the full length NPHS2 promoter - NPHS2(FL)-eGFP (see Figure 4A).
  • ⁇ A construct comprising the 5’ truncated NPHS2 promoter shown in SEQ ID NO: 2 - NPHS2(628bp)-eGFP (see Figure 4B).
  • the sequence of the full length NPHS2 promoter (NPHS2(FL)) used is shown in SEQ ID NO: 1.
  • the sequence of the 5’ truncated NPHS2 promoter (NPHS2(628bp)) used is shown in SEQ ID NO: 2.
  • HEK293T cells were seeded at a density of 1e6 cells per well of a 6-well plate and allowed to adhere overnight. Cells were cultured in DMEM supplemented with 10%FBS, in a total volume of 2mls. The following day cells were transfected as follows: 1ug of DNA was added to DMEM to a total volume of 150ul. In a separate tube, 3ul of PEI was added to 147ul DMEM. The PEI/DMEM was then added to the tube containing the DNA/DMEM and the solution was mixed by pipetting up and down ten times. This was incubated for 15 minutes at room temperature and then added dropwise to the well to be transfected.
  • NPHS2(FL)-eGFP see Figure 4A
  • NPHS2(628bp)-eGFP see Figure 4B
  • Materials & Methods Mouse podocytes were seeded at a density of 5e5 cells per well of a 6-well plate and allowed to adhere overnight. Cells were cultured in RPMI supplemented with 10%FBS and 1% ITS (Insulin, Transferrin & Selenium), in a total volume of 2mls. The following day cells were transfected as follows: 1ug of DNA was added to DMEM to a total volume of 150ul. In a separate tube, 3ul of PEI was added to 147ul DMEM.
  • the PEI/DMEM was then added to the tube containing the DNA/DMEM and the solution was mixed by pipetting up and down ten times. This was incubated for 15 minutes at room temperature and then added dropwise to the well to be transfected. Experiment was performed once with three technical replicates per sample. 48 hours following transfection, cells were analysed by FACS for GFP expression. Briefly, for each sample, media was removed from cells and cells were washed with 1ml of PBS. Following removal of PBS, 500ul of Trypsin was added to cells and incubated at 37 degrees for 5 minutes. Once cells were detached, trypsin was neutralised with 500ul DMEM supplemented with 10% FBS.
  • HEK293T cells were seeded at a density of 1e6 cells per well of a 6-well plate and allowed to adhere overnight. Cells were cultured in DMEM supplemented with 10%FBS, in a total volume of 2mls. The following day cells were transfected as follows: 1ug of DNA was added to DMEM to a total volume of 150ul. In a separate tube, 3ul of PEI was added to 147ul DMEM.
  • a luciferase assay was performed 48 hours following transfection using the Firefly Luciferase Glow Assay kit from Invitrogen (Cat. # 16177). Briefly, for each sample, media was removed from cells and cells were washed with 1ml of PBS. Following removal of PBS, 500ul of Trypsin was added to cells and incubated at 37 degrees for 5 minutes.
  • Each of the plasmids had increased luciferase activity compared to the non-transfected cells, demonstrating that each of the 5’ truncated podocin promoters was capable of driving luciferase expression.
  • the dotted line in Figure 5 indicates the mean luciferase activity of a plasmid comprising the control 628bp promoter (PS0471).
  • PS0576, PS0582, and PS0589 plasmids had significantly higher luciferase activity compared to PS0471 (one-way ANOVA).
  • Materials and methods AD293 Cell Culture AD293 cells were maintained in DMEM supplemented with 10% FBS. Cells were passaged at a ratio of 1:10 once confluency reached approximately 80%.
  • AD293 Transfections For transfections, cells were seeded at a density of 2e5 cells per well of a 6-well plate and allowed to adhere overnight. The following day transfections were set up as follows; a master mix of PEI/DMEM was made by mixing 120ul of PEI with 4.38mls of DMEM. For each plasmid, 2ug of DNA was mixed with DMEM to a final volume of 150ul. To each plasmid/DMEM mix, 150ul PEI mastermix was added and mixed 10 times by pipetting. This was incubated for 20 minutes at room temperature and then the total 300ul was added to a well of a 6-well plate. Media was changed the following morning, and cells harvested for luciferase assay a further 24 hours later.
  • An AAV vector comprising a transgene for use in gene therapy, wherein said AAV vector is administered to a subject, wherein the transgene is expressed in podocytes of said subject, and wherein the transgene is operably linked to a 5’-truncated podocin promoter.
  • an AAV vector comprising a transgene in the manufacture of a medicament for gene therapy, wherein said AAV vector is administered to a subject, wherein the transgene is expressed in podocytes of said subject, and wherein the transgene is operably linked to a 5’- truncated podocin promoter.
  • a method of expressing a transgene in podocytes of a subject comprising administering an AAV vector comprising the transgene to said subject, wherein the transgene is expressed in podocytes of said subject, and wherein the transgene is operably linked to a 5’-truncated podocin promoter.
  • VEGFC vascular endothelial growth factor
  • a complement protein such as CFI, CFH, FHL-1, C1INH, C4BP, MASP2, C3, C5aR1, C5, C5a, CD55, CD35, CD46, CD59, vitronectin, or clusterin.
  • 26. The AAV vector for use according to any of paras 1 or 4-25, the use according to any of paras 2 or 4-25, or the method according to any of paras 3 or 4-25, wherein the subject is suffering from or is at risk of a kidney disease or a glomerular disease.
  • An AAV vector comprising a therapeutic transgene, wherein the transgene is operably linked to a 5’-truncated podocin promoter, and wherein the AAV vector does not comprise: (i) a nephrotic syndrome-associated transgene, such as NPHS2, ADCK4, ALG1, ARHGAP24, ARGHDIA, CD151, CD2AP, COQ2, COQ6, DGKE, E2F3, EMP2, KANK2, LAGE3, LMNA, LMX1B, MAFB, NUP85, NUP93, NXF5, OSGEP, PAX2, PDSS2, PMM2, PODXL, SCARB2, SGPL1, Smad7, TP53RK, TPRKB, VDR, WDR73, WT1, ZMPSTE24, APOL1, NPHS1, TRPC6, NUP107, NUP133, NUP160, ACTN4, INF2, ANKFY1, ANLN, CRB2, ITGA3, KANK1, KANK4, MA
  • the transgene is operably linked to a Woodchuck hepatitis post-transcriptional regulatory element (WPRE).
  • 43. A cell comprising the AAV vector according to any of paras 33-42.
  • 44. A pharmaceutical composition comprising the AAV vector according to any of paras 33- 42, or the cell according to para 43.
  • the promoter according to para 46 or 47, wherein the promoter consists of the nucleotide sequence of SEQ ID NO: 2.
  • 49. A polynucleotide comprising the promoter according to any of paras 46-48. 50. The polynucleotide according to para 49, wherein the promoter is operably linked to a transgene.
  • 51. The polynucleotide according to para 49 or 50, wherein the transgene is operably linked to one or more further regulatory elements, such as a post-transcriptional regulatory element and/or a polyadenylation sequence.
  • 52. A vector comprising the polynucleotide according to any of paras 49-51. 53.
  • the vector according to para 52 wherein the vector is capable of transducing podocytes, optionally wherein the vector is capable of specifically transducing podocytes.
  • the vector is a viral vector, such as an adeno-associated virus (AAV) vector, a lentiviral vector, a retroviral vector, an adenoviral vector, an adeno-associated viral vector, a herpes simplex viral vector, an alphaviral vector, a flaviviral vector, a rhabdoviral vector, a measles viral vector, a Newcastle disease viral vector, a poxviral vector, and a picornaviral vector, preferably wherein the vector is an AAV vector.
  • AAV adeno-associated virus
  • the vector according to para 54 wherein the viral vector is in the form of a viral vector particle, preferably wherein the viral vector is in the form of an AAV vector particle.
  • 56. The vector according to any of paras 59-62, wherein the vector is the form of an AAV vector particle encapsidated by AAV3B, LK03, AAV9, ShH10, AAV-DJ, AAV2, AAV6.2, or AAV5 capsid proteins.
  • a cell comprising the polynucleotide according to any of paras 49-51, or the vector according to any of paras 52-56. 58.
  • a pharmaceutical composition comprising the polynucleotide according to any of paras 49-51, the vector according to any of paras 52-56, or the cell according to para 57.

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Abstract

An AAV vector comprising a transgene for use in gene therapy, wherein said AAV vector is administered to a subject, wherein the transgene is expressed in podocytes of said subject, and wherein the transgene is operably linked to a 5'-truncated podocin promoter.

Description

PROMOTER FIELD OF THE INVENTION The present invention relates to promoters which are capable of driving transgene expression in podocytes. The present invention also relates to vectors comprising said promoters and their use in therapy. BACKGROUND TO THE INVENTION There are many diseases that affect kidney function by attacking the glomerulus. The glomerulus filters approximately 180 litres of plasma each day, and the healthy glomerular filtration barrier has an astonishing ability to retain about 99.9% of large proteins including albumin over our lifetimes without clogging. The glomerular filtration barrier (GFB) comprises 3 main layers: the glomerular endothelial cell, the glomerular basement membrane (GBM) and the podocyte. The GBM is made of a highly cross-linked macromolecular meshwork of type IV collagen, proteoglycans, and laminin. Genetic forms of glomerular disease can be caused by genetic defects in these molecular structures. For example, Alport syndrome is caused by pathogenic variants in the COL4A3, COL4A4 and COL4A5 genes, which result in abnormalities of the collagen IV α345 network of basement membranes. Alport syndrome affects approximately 1 in 5,000-10,000 of all individuals in continental Europe and the USA. The condition usually presents during childhood and is associated with a spectrum of phenotypes that include a progressive loss of kidney function, and can include hearing loss and eye abnormalities. Other GBM-associated diseases include Pierson syndrome and Nail-patella syndrome (Chiang, C.K. and Inagi, R., 2010. Nature Reviews Nephrology, 6(9), p.539). The podocyte has also been implicated as a key cell in the progression of glomerular disease. Podocytes are mesodermally derived cells that are highly specialized and found only in the renal glomerulus. They exhibit unique characteristics such as foot processes and slit diaphragms, which are critical for glomerular filtration. Podocyte-associated genetic glomerular diseases include Nephrotic Syndrome, Frasier syndrome and Denys–Drash syndrome, Schimke immuno-osseous dysplasia, and Epstein and Fechtner syndrome. (Chiang, C.K. and Inagi, R., 2010. Nature Reviews Nephrology, 6(9), p.539). Accordingly, glomerular cells, such as podocytes, represent a potential target for gene therapy approaches. In order to maximise gene therapy potential, promoter sequences which can drive transgene expression in glomerular cells, such as podocytes, are required. Wong et al. (American Journal of Physiology Renal Physiology; 2000; 279(6); F1027-32) described a 1.25-kb DNA fragment from the human nephrin promoter and 5′-flanking region that is capable of directing podocyte-specific expression. This promoter has been used to achieve kidney-specific expression of GFP using an AAV9 vector (Picconi et al.; 2014; Molecular Therapy – Methods & Clinical Development; 1, 14014). At present, a significant amount of vector cargo capacity (e.g. AAV cargo capacity) is taken up with non-coding elements such as the nephrin or podocin promotors, WPRE elements and polyadenylation sequence. Thus, there is a need in the art for minimal promoters which can drive transgene expression in glomerular cells, such as podocytes, in order to provide maximal flexibility for gene therapy approaches for glomerular diseases. SUMMARY OF THE INVENTION The present invention is based on the inventors surprising provision of a minimal podocin promoter which is capable of driving transgene expression in, in particular, kidney cells such as podocytes. According to a first aspect of the present invention there is provided an AAV vector comprising a transgene for use in therapy, wherein said AAV vector is administered to a subject, wherein the transgene is expressed in podocytes of said subject, and wherein the transgene is operably linked to a 5’-truncated podocin promoter. According to another aspect of the present invention there is provided use of an AAV vector comprising a transgene in the manufacture of a medicament for therapy, wherein said AAV vector is administered to a subject, wherein the transgene is expressed in podocytes of said subject, and wherein the transgene is operably linked to a 5’-truncated podocin promoter. Preferably the therapy is gene therapy. According to another aspect of the present invention there is provided a method of expressing a transgene in podocytes of a subject, the method comprising administering an AAV vector comprising the transgene to said subject, wherein the transgene is expressed in podocytes of said subject, and wherein the transgene is operably linked to a 5’-truncated podocin promoter. The 5’-truncated podocin promoter may be a truncate of a full-length podocin promoter wherein the full-length podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 1. In various embodiments, the 5’-truncated podocin promoter has a length of about 1.5 kb or less, about 1.4 kb or less, about 1.3 kb or less, about 1.2 kb or less, about 1.1 kb or less, about 1.0 kb or less, about 0.9 kb or less, about 0.8 kb or less, about 0.7 kb or less, or about 0.6 kb or less. In one embodiment, the 5’-truncated podocin promoter has a length of 628 base pairs (bp) or less, 600 bp or less, 550 bp or less, 500 bp or less, 450 bp or less, 400 bp or less, 350 bp or less, 300 bp or less, 250 bp or less, or 200 bp or less. In one embodiment, the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to any of SEQ ID NOs: 2 or 35-54. In one embodiment, the 5’-truncated podocin promoter has a length of about 628 base pairs (bp). In one embodiment, the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 2. In one embodiment, the 5’-truncated podocin promoter has a length of about 600 base pairs (bp). In one embodiment, the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 36. In one embodiment, the 5’-truncated podocin promoter has a length of about 578 base pairs (bp). In one embodiment, the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 47. In one embodiment, the 5’-truncated podocin promoter has a length of about 550 base pairs (bp). In one embodiment, the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 37. In one embodiment, the 5’-truncated podocin promoter has a length of about 528 base pairs (bp). In one embodiment, the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 48. In one embodiment, the 5’-truncated podocin promoter has a length of about 500 base pairs (bp). In one embodiment, the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 38. In one embodiment, the 5’-truncated podocin promoter has a length of about 478 base pairs (bp). In one embodiment, the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 49. In one embodiment, the 5’-truncated podocin promoter has a length of about 450 base pairs (bp). In one embodiment, the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 39. In one embodiment, the 5’-truncated podocin promoter has a length of about 428 base pairs (bp). In one embodiment, the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 50. In one embodiment, the 5’-truncated podocin promoter has a length of about 400 base pairs (bp). In one embodiment, the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 40. In one embodiment, the 5’-truncated podocin promoter has a length of about 378 base pairs (bp). In one embodiment, the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 51. In one embodiment, the 5’-truncated podocin promoter has a length of about 350 base pairs (bp). In one embodiment, the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 41. In one embodiment, the 5’-truncated podocin promoter has a length of about 328 base pairs (bp). In one embodiment, the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 52. In one embodiment, the 5’-truncated podocin promoter has a length of about 300 base pairs (bp). In one embodiment, the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 42. In one embodiment, the 5’-truncated podocin promoter has a length of about 278 base pairs (bp). In one embodiment, the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 53. In one embodiment, the 5’-truncated podocin promoter has a length of about 250 base pairs (bp). In one embodiment, the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 43. In one embodiment, the 5’-truncated podocin promoter has a length of about 228 base pairs (bp). In one embodiment, the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 54. In one embodiment, the 5’-truncated podocin promoter has a length of about 200 base pairs (bp). In one embodiment, the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 44. In one embodiment, the 5’-truncated podocin promoter has a length of about 150 base pairs (bp). In one embodiment, the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 45. The AAV vector used in the present invention is preferably administered to the subject in the form of an AAV vector particle. The AAV vector particle may be encapsidated by AAV3B, LK03, AAV9, ShH10, AAV-DJ, AAV2, AAV6.2, or AAV5 capsid proteins. In one embodiment, the AAV vector particle is encapsidated by AAV3B, LK03, or AAV9 capsid proteins. In one embodiment, the AAV vector particle is encapsidated by LK03 capsid proteins. In one embodiment, the AAV vector particle is encapsidated by AAV9 capsid proteins. In one embodiment, the AAV vector particle is not encapsidated by AAV8 capsid proteins or AAV9 capsid proteins. In one embodiment, the AAV vector particle is encapsidated by ShH10, AAV-DJ, AAV2, AAV6.2, or AAV5 capsid proteins. Preferably, the transgene used in the present invention is a therapeutic transgene. Preferably, the AAV vector does not comprise a reporter transgene, such as LacZ. In one embodiment, the AAV vector does not comprise a nephrotic syndrome-associated transgene, such as NPHS2, ADCK4, ALG1, ARHGAP24, ARGHDIA, CD151, CD2AP, COQ2, COQ6, DGKE, E2F3, EMP2, KANK2, LAGE3, LMNA, LMX1B, MAFB, NUP85, NUP93, NXF5, OSGEP, PAX2, PDSS2, PMM2, PODXL, SCARB2, SGPL1, Smad7, TP53RK, TPRKB, VDR, WDR73, WT1, ZMPSTE24, APOL1, NPHS1, TRPC6, NUP107, NUP133, NUP160, ACTN4, INF2, ANKFY1, ANLN, CRB2, ITGA3, KANK1, KANK4, MAGI2, MYO1E, OCRL, PTPRO, SMARCAL1, SYNPO, TBC1D8B, XPO5, TNS2 or NLRP3. In one embodiment, the AAV vector does not comprise a vascular endothelial growth factor (VEGFC) transgene. In one embodiment, the AAV vector does not comprise a COL4A3, COL4A4, or COL4A5 transgene. In one embodiment, the AAV vector does not comprise a nucleotide sequence encoding a complement protein, such as CFI, CFH, FHL-1, C1INH, C4BP, MASP2, C3, C5aR1, C5, C5a, CD55, CD35, CD46, CD59, vitronectin, or clusterin. In one embodiment, the AAV vector does not comprise a NPHS2, ADCK4, ALG1, ARHGAP24, ARGHDIA, CD151, CD2AP, COQ2, COQ6, DGKE, E2F3, EMP2, KANK2, LAGE3, LMNA, LMX1B, MAFB, NUP85, NUP93, NXF5, OSGEP, PAX2, PDSS2, PMM2, PODXL, SCARB2, SGPL1, Smad7, TP53RK, TPRKB, VDR, WDR73, WT1, ZMPSTE24, APOL1, NPHS1, TRPC6, NUP107, NUP133, NUP160, ACTN4, INF2, ANKFY1, ANLN, CRB2, ITGA3, KANK1, KANK4, MAGI2, MYO1E, OCRL, PTPRO, SMARCAL1, SYNPO, TBC1D8B, XPO5, TNS2. NLRP3, VEGFC, COL4A3, COL4A4, COL4A5, CFI, CFH, FHL-1, C1INH, C4BP, MASP2, C3, C5aR1, C5, C5a, CD55, CD35, CD46, CD59, vitronectin, or clusterin transgene. According to the present invention, the transgene may be operably linked to a Woodchuck hepatitis post-transcriptional regulatory element (WPRE). According to the present invention, the transgene may be operably linked to a polyadenylation signal. According to the present invention, the transgene may be operably linked to a Kozak sequence. In a preferred embodiment of the present invention, the expression of the transgene in the podocytes of a subject administered the AAV vector defined herein treats or prevents a kidney disease or a glomerular disease in said subject. The subject may have a defective gene corresponding to said transgene. Preferably, the subject is a human subject. Preferably, the subject is suffering from or is at risk of a kidney disease or a glomerular disease. The AAV vector may be administered in the form of a pharmaceutical composition comprising a pharmaceutically acceptable carrier, diluent or excipient. The AAV vector may be administered systemically and/or by intravenous injection. The AAV vector may be administered locally and/or by injection into the renal artery. The AAV vector may be administered in a dose of from about 1x106 vg/kg to about 1x1014 vg/kg, from about 1x106 vg/kg to about 1x1013 vg/kg, or from about 1x109 vg/kg to about 1x1012 vg/kg. The administration of the AAV vector may result in the transgene being specifically expressed in podocytes. In one embodiment, the transgene is expressed in 5% or more, 10% or more, 15% or more, 20% or more, 30% or more or 40% or more podocytes in at least one of the subject’s kidneys. According to another aspect of the present invention there is provided an AAV vector comprising a therapeutic transgene, wherein the transgene is operably linked to a 5’-truncated podocin promoter as defined herein, and wherein the AAV vector does not comprise: (i) a nephrotic syndrome-associated transgene, such as NPHS2, ADCK4, ALG1, ARHGAP24, ARGHDIA, CD151, CD2AP, COQ2, COQ6, DGKE, E2F3, EMP2, KANK2, LAGE3, LMNA, LMX1B, MAFB, NUP85, NUP93, NXF5, OSGEP, PAX2, PDSS2, PMM2, PODXL, SCARB2, SGPL1, Smad7, TP53RK, TPRKB, VDR, WDR73, WT1, ZMPSTE24, APOL1, NPHS1, TRPC6, NUP107, NUP133, NUP160, ACTN4, INF2, ANKFY1, ANLN, CRB2, ITGA3, KANK1, KANK4, MAGI2, MYO1E, OCRL, PTPRO, SMARCAL1, SYNPO, TBC1D8B, XPO5, TNS2 or NLRP3; (ii) a vascular endothelial growth factor (VEGFC) transgene; (iii) a COL4A3, COL4A4, or COL4A5 transgene; or (iv) a nucleotide sequence encoding a complement protein, such as CFI, CFH, FHL-1, C1INH, C4BP, MASP2, C3, C5aR1, C5, C5a, CD55, CD35, CD46, CD59, vitronectin, or clusterin. According to another aspect of the present invention there is provided an AAV vector particle encapsidated by AAV9 capsid proteins and comprising a therapeutic transgene, wherein the transgene is operably linked to a 5’-truncated podocin promoter as defined herein. According to another aspect of the present invention there is provided an AAV vector particle encapsidated by LK03 capsid proteins and comprising a therapeutic transgene, wherein the transgene is operably linked to a 5’-truncated podocin promoter as defined herein. According to another aspect of the present invention there is provided an AAV vector particle encapsidated by ShH10, AAV-DJ, AAV2, AAV6.2, or AAV5 capsid proteins and comprising a therapeutic transgene, wherein the transgene is operably linked to a 5’-truncated podocin promoter as defined herein. According to another aspect of the present invention there is provided a cell comprising the AAV vector defined herein. According to another aspect of the present invention there is provided a pharmaceutical composition comprising the AAV vector or the cell defined herein. According to another aspect of the present invention there is provided a promoter consisting of a nucleotide sequence having at least 97.8% identity to SEQ ID NO: 2. In one embodiment, the promoter consists of a nucleotide sequence having at least 97.9%, at least 98.0%, at least 98.1%, at least 98.2%, at least 98.3%, at least 98.4%, at least 98.5%, at least 98.6%, at least 98.7%, at least 98.8%, at least 98.9%, at least 99.0%, at least 99.1%, at least 99.2%, at least 99.3%, at least 99.4%, at least 99.5%, at least 99.6%, at least 99.7%, at least 99.8%, or at least 99.9% identity to SEQ ID NO: 2. In one embodiment, the promoter consists of the nucleotide sequence of SEQ ID NO: 2. According to another aspect of the present invention there is provided a 5’-truncated podocin promoter having a length of 600bp or less. In one embodiment, the 5’-truncated podocin promoter has a length of 550bp or less, 500bp or less, 450bp or less, 400bp or less, 350 bp or less, 300 bp or less, 250 bp or less, or 200 bp or less. The 5’-truncated podocin promoter may be a truncate of a full-length podocin promoter wherein the full-length podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 1. In one embodiment, the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to any of SEQ ID NOs: 36-54. According to another aspect of the present invention there is provided a promoter which consists of a nucleotide sequence having at least 90% identity to any of SEQ ID NOs: 36-54. In one embodiment, the promoter consists of a nucleotide sequence having at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to any of SEQ ID NOs: 36-54. In one embodiment, the promoter consists of a nucleotide sequence having at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to any of SEQ ID NOs: 37-46 or 47-54, any of SEQ ID NOs: 37-46 or 48-54, any of SEQ ID NOs: 38-46 or 48-54, any of SEQ ID NOs: 38- 46 or 49-54, any of SEQ ID NOs: 39-46 or 49-54, any of SEQ ID NOs: 39-46 or 50-54, any of SEQ ID NOs: 40-46 or 50-54, any of SEQ ID NOs: 40-46 or 51-54, any of SEQ ID NOs: 41- 46 or 51-54, any of SEQ ID NOs: 41-46 or 52-54, any of SEQ ID NOs: 42-46 or 52-54, any of SEQ ID NOs: 42-46 or 53-54, any of SEQ ID NOs: 43-46 or 53-54, or any of SEQ ID NOs: 43- 46 or 54. In one embodiment, the promoter consists of a nucleotide sequence having at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to any of SEQ ID NOs: 38, 40, 41, 42, 44, 47, 48, 51 or 53. In one embodiment, the promoter consists of a nucleotide sequence having at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to any of SEQ ID NOs: 40, 47 or 53. According to another aspect of the present invention there is provided a polynucleotide comprising the promoter as defined herein. In one embodiment, the promoter is operably linked to a transgene. In one embodiment, the transgene is operably linked to one or more further regulatory elements, such as a post- transcriptional regulatory element and/or a polyadenylation sequence. According to another aspect of the present invention there is provided a vector comprising the polynucleotide as defined herein. In one embodiment, the vector is capable of transducing podocytes, optionally wherein the vector is capable of specifically transducing podocytes. Suitably, the vector is a viral vector, such as an adeno-associated virus (AAV) vector, a lentiviral vector, a retroviral vector, an adenoviral vector, an adeno-associated viral vector, a herpes simplex viral vector, an alphaviral vector, a flaviviral vector, a rhabdoviral vector, a measles viral vector, a Newcastle disease viral vector, a poxviral vector, or a picornaviral vector, preferably wherein the vector is an AAV vector. In one embodiment, the viral vector is in the form of a viral vector particle, preferably wherein the viral vector is in the form of an AAV vector particle. In one embodiment, the vector is the form of an AAV vector particle encapsidated by AAV3B, LK03, AAV9, ShH10, AAV-DJ, AAV2, AAV6.2, or AAV5 capsid proteins. According to another aspect of the present invention there is provided a cell comprising the polynucleotide, or the vector as defined herein. According to another aspect of the present invention there is provided a pharmaceutical composition comprising the polynucleotide, the vector, or the cell as defined herein. According to another aspect of the present invention there is provided the polynucleotide according, the vector, or the cell as defined herein, for use in medicine, optionally for use in a method as defined herein. According to another aspect of the present invention there is provided use of the promoter as defined herein to drive expression of a transgene. The expression may be kidney-specific. DESCRIPTION OF DRAWINGS Figure 1: Transfection of HEK293T cells with FL (full-length) and 628bp promoters driving GFP expression. Figure 2: Transfection of mouse podocytes with FL (full-length) and 628bp promoters driving GFP expression. Figure 3: Transfection of HEK293T cells with FL (full-length) and 628bp promoters driving Luciferase expression compared to non-transfected (NT) cells. Figure 4: Schematic of constructs used in Examples 1-3. (A) NPSH2(FL)-eGFP construct used in Examples 1 and 2. (B) NPSH2(628)-eGFP construct used in Examples 1 and 2. (C) NPHS2(FL)-Luciferase-GFP construct used in Example 3. (C) NPHS2(628)-Luciferase-GFP construct used in Example 3. Figure 5: Luciferase activity mediated by truncated NPHS2 promoters (n=3). AD293 cells were transfected with plasmids comprising a luciferase transgene under the control of truncated podocin promoters having lengths of from 150 bp to 600 bp (PS0576 to PS0593). Luciferase activity is compared to non-transfected cells and 628bp promoter (PS0471). DETAILED DESCRIPTION Various preferred features and embodiments of the present invention will now be described by way of non-limiting examples. It must be noted that as used herein and in the appended claims, the singular forms "a", "an", and "the" include plural referents unless the context clearly dictates otherwise. The terms "comprising", "comprises" and "comprised of" as used herein are synonymous with "including", "includes" or "containing", "contains", and are inclusive or open-ended and do not exclude additional, non-recited members, elements or method steps. The terms "comprising", "comprises" and "comprised of" also include the term "consisting of". The publications discussed herein are provided solely for their disclosure prior to the filing date of the present application. Nothing herein is to be construed as an admission that such publications constitute prior art to the claims appended hereto. This disclosure is not limited by the exemplary methods and materials disclosed herein, and any methods and materials similar or equivalent to those described herein can be used in the practice or testing of embodiments of this disclosure. Numeric ranges are inclusive of the numbers defining the range. Unless otherwise indicated, any nucleic acid sequences are written left to right in 5' to 3' orientation; amino acid sequences are written left to right in amino to carboxy orientation, respectively. The practice of the present invention will employ, unless otherwise indicated, conventional techniques of chemistry, biochemistry, molecular biology, microbiology and immunology, which are within the capabilities of a person of ordinary skill in the art. Such techniques are explained in the literature. See, for example, Sambrook, J., Fritsch, E.F. and Maniatis, T. (1989) Molecular Cloning: A Laboratory Manual, 2nd Edition, Cold Spring Harbor Laboratory Press; Ausubel, F.M. et al. (1995 and periodic supplements) Current Protocols in Molecular Biology, Ch.9, 13 and 16, John Wiley & Sons; Roe, B., Crabtree, J. and Kahn, A. (1996) DNA Isolation and Sequencing: Essential Techniques, John Wiley & Sons; Polak, J.M. and McGee, J.O’D. (1990) In Situ Hybridization: Principles and Practice, Oxford University Press; Gait, M.J. (1984) Oligonucleotide Synthesis: A Practical Approach, IRL Press; and Lilley, D.M. and Dahlberg, J.E. (1992) Methods in Enzymology: DNA Structures Part A: Synthesis and Physical Analysis of DNA, Academic Press. Each of these general texts is herein incorporated by reference Promoters A “promoter” is used herein according to its typical meaning to refer to a sequence of DNA which regulates and/or initiates transcription of RNA from a DNA sequence. Suitably, the promoter is operable in mammalian cells, e.g. human cells. The promoter may be operable in kidney cells. The promoter of the invention may be capable of driving transgene expression in the kidney. Examples of kidney cells in which the promoter may be operable include, but are not limited to, glomerular cells. The promoter of the invention may be capable of driving transgene expression in the glomerulus. The mature glomerulus contains four cell types: Parietal epithelial cells that form Bowman's capsule, podocytes that cover the outermost layer of the glomerular filtration barrier, glycocalyx-coated fenestrated endothelial cells that are in direct contact with blood, and mesangial cells that sit between the capillary loops (Vaughan, M.R. and Quaggin, S.E., 2008. Journal of the American Society of Nephrology, 19(1), pp.24-33). The promoter may be operable in a podocyte cell. The promoter of the invention may be capable of driving transgene expression in podocytes. The promoter of the invention may be a tissue-specific promoter. As used herein, a “tissue- specific promoter” is a promoter which preferentially facilitates expression of a transgene in a specific type of cells or tissue. Suitably, a tissue-specific promoter may facilitate higher expression of a transgene in one cell-type as compared to other cell-types. For example, a tissue-specific promoter may be a promoter which facilitates transgene expression levels at least 10% higher, at least 20% higher, at least 30% higher, at least 40% higher, at least 50% higher, at least 100% higher, at least 200% higher, at least 300% higher, at least 400% higher, at least 500% higher, or at least 1000% higher in one cell-type as compared to expression levels in other cell-types. Suitably, the promoter is a kidney-specific promoter. In some embodiments, the promoter is a podocyte-specific promoter. Transgene expression may be measured by any suitable method known in the art. For example by measuring the expression of a reporter transgene, e.g. Green fluorescent protein (GFP), operably linked to the promoter, wherein expression of the reporter transgene correlates with the ability of the promoter to facilitate expression of a gene. Expression of the reporter transgene, e.g. GFP may be determined by any suitable method e.g. FACS. For example, a kidney-specific promoter may facilitate higher expression of a reporter transgene in kidney cells compared to other cell types (e.g. CNS, retina, lung, pancreas, heart, or muscle cells). For example, a podocyte-specific promoter may facilitate higher expression of a reporter transgene in conditionally immortalised podocytes compared to other cell-types. Suitable podocyte cell lines will be well known to those of skill in the art, for example CIHP-1. Methods to generate immortalized podocytes will be well known to those of skill in the art. Suitable methods are described in Ni, L., et al., 2012. Nephrology, 17(6), pp.525-531. The 5’-truncated podocin promoter used in the invention is a truncate of a full-length podocin promoter. Preferably the full-length podocin promoter sequence has at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 1 shown below. SEQ ID NO: 1 GCCCTCCTATTTAGTCTCTCTGCCACCTACAAATTGAGAAAGTCAAATTTAGTAAGTCCT TATAGTTTCCAGCTCTAAAACAACAGGATTTGTAACATCATGTAGTGCCAATCAATTCCA GCCTTTCCATTAAATAATCCAAATGAATCAGTATTCATGCATTCACTTATTCATATGGATA TAATACTTCAAAACACTAACTGGGAATAAGTGTAATACTTTAGAAAGCCTTGCACTCTTT CATAATCTTTCACCTTGGAAACATAAAAGTAAACAGCTTTAATTGATCCTCCCTTTCTTCT AGTAACCCAGTTCCCACAGATGTCAGTTTCTTAACAAATCCAAACAAGGAGTAAAGTATT GAGAGTTTCAGAATGTGTGCATATCATATTTTCAGCAAAGGGATGAGCAACACTTTCAAT TAAAAAGGAATCTAAAAGAAAACAGCAGCATCCTTGTAAAAGTAGATGAGGACAGCCAG CTGGACTTAGCTGCTAACTCAGCACTCCCTTCCCCAACTCCTCCACCTTCCAGCCATGC TGCATTGACCTTTTTATCATGGAATGGTATGCAGCTTCTCTGATTTTGTCATTGTCACTC ATAATCCCAGCAGACTTGTGTTATAAGCAGCTGCTTATATTGCTGTTATGTATAGATATA GAAATGTAACTTGTCAGTGGTCAAAGAAGCCAAGGAAACTGAGCAAAGGTGACTGAGA ACAAGTGCTCAGCATGGGCTCCAACACCCAGTAGGGCCTTTTCTCATTGATTTTTCTCC TACAAGGAATTTCTCTCTTATTGAAAGACAGTACAGTTTGGGGAATATCATTTGACCAGA TATCCTGCTCCATTCTTGGAATGAAAAACAGGTGCAATTTTAGTATAAGAGTCTAGCTAG GGATGCGGATGAAATTAATTTATAACAGGACCAGGCTGAGGAAAAACACCAATAAATGC TACAAATAGTTTGTCACTTTTTCTAACAGGGAAATCTCTAAAGGATGATAAAGATGCTAG CTGTGTTTAATAGCAAATGTTAATTTGGGGGAATATGAAATAATCATGATATCTTTCTCTC TTTGAGCCAAACGCAGCTTTGGAATTTTTCTCACTGTAGTGTTGTAGGCAGAAACTATCA ACTGAGGCAGGCCCTTGCGAGGAAATATTTATCCTCCTTAGCCCATGCAATGATCAGGT TTAGACAGTAAAAAATCTATTTAAGTGACTGGATTGCTCATCTTTTATTTACTTTACTCCC AGTGGCCTAACCTGGAACATATAGAAGAAAGTAGTGGAATGGTTTTTCCAGACGCACTT CAGTTACTTCAGGTCCTCAGTGTTTAATAAGGTTTTGGGAGAGAAAAAAAGCTATTGCT GTGTGAATTCAATAAATATTTAAAAAATTTAAAAAATCAGTGAGCATTATTTCTGCCATCA TGTGATCTGAACTATCTGAGTAGTATCATAAATCTTGGGTGATGGGTTACCTTGACCAAC CTCCCTTTCAGTAAGAAACAAATTATTAACAGAAAGTGAAAGAGAAATAAACCTATTCAA TGTTTCAAGTCCCTCAGAAGAGGGTGAGGCAGCTCCCCAGTTCGTTGCTAGATCCAGC CTGGCCAATGAACCCTGAAAAAGCCCAACTCCTGCTTTCATCATGGAAGCACGGGACA AAGTGTCTCTTCCTAAAAACAGAAGTTAGACCAGACCCCTTCCTGCCTATGATTCTTCAA GAAGCATTGCATCATCAACATCAGGCATAAGCATTAATAAAGACCCTAAATAATAACAGA GACGAAACACATCGCAAAGAGAGTTTTCTTTTATCCCCTTTAAAATGTAAATACTCCCAG GAGGAATCAGCCAACATCATTAGGGGTTAATGCATATGTAGAATAACTAGGGCCAGGAT ATAAAATAAGAAATACGTAGGGAGGAGAGAAAGGCATCCTTGAGACGACTCCAAGAAG GAAAGTTGGGGATGAGGCGAAATTTCTGATTTTACCTTAAAGTGACCCTAATTCGATGA CCTTTTGTGGTTTTTTTCTTTTTTCTTTTTTACTTGGCCCTGCCCAAGCAGGACCTAAAAA CAAACAGACAAAAAAGGTTACTAACAACTGTTCCTCTCCACGAAAATCTGCAGTAAAAG GTAAAAGATGTATTCGTTTTGAAGAGAAACCAGAGCTTGCGATGAGCTTCTGTATCTCC GTCAGCCCTCTAGCATGACATTAGGAACCCTCCAGGAGATGAGTCTTCACAGCCCGGG TTGGCACCTGCAGACACGCACTTTTCAACGCCCGCACCCTGCCCGGGGCCGGCTCTC CCACCCAGGCCTCTCTCTGCTTCAGCGCCGCCCCGGCCGTGGGAGTCGGCGGGCGC AGTCCACAGCTCCACCAAGACACAGCTGTCGGGGTTCCGGGTGCGCCCCGCCCGCGG CCCCGGTGTCCCGCCCCTCGCCCTCAGCCCCCACCCGATGGTCTTTAGGGTCCCCCG GGCACGCCACGCGGACCCGCAGCGACTCCACAGGGACTGCGCTCCCGTGCCCCTAG CGCTCCCGCGCTGCTGCTCCAGCCGCCCGGCAGCTCTGACCATG In one embodiment, the 5’-truncated podocin promoter consists of nucleotides 1962-2589, 1963-2589, 1964-2589, 1965-2589, 1966-2589, 1967-2589, 1968-2589, 1969-2589, 1970- 2589, 1971-2589, 1972-2589, 1973-2589, 1974-2589, 1975-2589, 1976-2589, 1977-2589, 1978-2589, 1979-2589, 1980-2589, 1981-2589, 1982-2589, 1983-2589, 1984-2589, 1985- 2589, 1986-2589, 1987-2589, 1988-2589, 1989-2589, 1990-2589, 1991-2589, 1992-2589, 1993-2589, 1994-2589, 1995-2589, 1996-2589, 1997-2589, 1998-2589, 1999-2589, 2000- 2589, 2001-2589, 2002-2589, 2003-2589, 2004-2589, 2005-2589, 2006-2589, 2007-2589, 2008-2589, 2009-2589, 2010-2589, 2011-2589, 2012-2589, 2013-2589, 2014-2589, 2015- 2589, 2016-2589, 2017-2589, 2018-2589, 2019-2589, 2020-2589, 2021-2589, 2022-2589, 2023-2589, 2024-2589, 2025-2589, 2026-2589, 2027-2589, 2028-2589, 2029-2589, 2030- 2589, 2031-2589, 2032-2589, 2033-2589, 2034-2589, 2035-2589, 2036-2589, 2037-2589, 2038-2589, 2039-2589, 2040-2589, 2041-2589, 2042-2589, 2043-2589, 2044-2589, 2045- 2589, 2046-2589, 2047-2589, 2048-2589, 2049-2589, 2050-2589, 2051-2589, 2052-2589, 2053-2589, 2054-2589, 2055-2589, 2056-2589, 2057-2589, 2058-2589, 2059-2589, 2060- 2589, 2061-2589, 2062-2589, 2063-2589, 2064-2589, 2065-2589, 2066-2589, 2067-2589, 2068-2589, 2069-2589, 2070-2589, 2071-2589, 2072-2589, 2073-2589, 2074-2589, 2075- 2589, 2076-2589, 2077-2589, 2078-2589, 2079-2589, 2080-2589, 2081-2589, 2082-2589, 2083-2589, 2084-2589, 2085-2589, 2086-2589, 2087-2589, 2088-2589, 2089-2589, 2090- 2589, 2091-2589, 2092-2589, 2093-2589, 2094-2589, 2095-2589, 2096-2589, 2097-2589, 2098-2589, 2099-2589, 2100-2589, 2101-2589, 2102-2589, 2103-2589, 2104-2589, 2105- 2589, 2106-2589, 2107-2589, 2108-2589, 2109-2589, 2110-2589, 2111-2589, 2112-2589, 2113-2589, 2114-2589, 2115-2589, 2116-2589, 2117-2589, 2118-2589, 2119-2589, 2120- 2589, 2121-2589, 2122-2589, 2123-2589, 2124-2589, 2125-2589, 2126-2589, 2127-2589, 2128-2589, 2129-2589, 2130-2589, 2131-2589, 2132-2589, 2133-2589, 2134-2589, 2135- 2589, 2136-2589, 2137-2589, 2138-2589, 2139-2589, 2140-2589, 2141-2589, 2142-2589, 2143-2589, 2144-2589, 2145-2589, 2146-2589, 2147-2589, 2148-2589, 2149-2589, 2150- 2589, 2151-2589, 2152-2589, 2153-2589, 2154-2589, 2155-2589, 2156-2589, 2157-2589, 2158-2589, 2159-2589, 2160-2589, 2161-2589, 2162-2589, 2163-2589, 2164-2589, 2165- 2589, 2166-2589, 2167-2589, 2168-2589, 2169-2589, 2170-2589, 2171-2589, 2172-2589, 2173-2589, 2174-2589, 2175-2589, 2176-2589, 2177-2589, 2178-2589, 2179-2589, 2180- 2589, 2181-2589, 2182-2589, 2183-2589, 2184-2589, 2185-2589, 2186-2589, 2187-2589, 2188-2589, 2189-2589, 2190-2589, 2191-2589, 2192-2589, 2193-2589, 2194-2589, 2195- 2589, 2196-2589, 2197-2589, 2198-2589, 2199-2589, 2200-2589, 2201-2589, 2202-2589, 2203-2589, 2204-2589, 2205-2589, 2206-2589, 2207-2589, 2208-2589, 2209-2589, 2210- 2589, 2211-2589, 2212-2589, 2213-2589, 2214-2589, 2215-2589, 2216-2589, 2217-2589, 2218-2589, 2219-2589, 2220-2589, 2221-2589, 2222-2589, 2223-2589, 2224-2589, 2225- 2589, 2226-2589, 2227-2589, 2228-2589, 2229-2589, 2230-2589, 2231-2589, 2232-2589, 2233-2589, 2234-2589, 2235-2589, 2236-2589, 2237-2589, 2238-2589, 2239-2589, 2240- 2589, 2241-2589, 2242-2589, 2243-2589, 2244-2589, 2245-2589, 2246-2589, 2247-2589, 2248-2589, 2249-2589, 2250-2589, 2251-2589, 2252-2589, 2253-2589, 2254-2589, 2255- 2589, 2256-2589, 2257-2589, 2258-2589, 2259-2589, 2260-2589, 2261-2589, 2262-2589, 2263-2589, 2264-2589, 2265-2589, 2266-2589, 2267-2589, 2268-2589, 2269-2589, 2270- 2589, 2271-2589, 2272-2589, 2273-2589, 2274-2589, 2275-2589, 2276-2589, 2277-2589, 2278-2589, 2279-2589, 2280-2589, 2281-2589, 2282-2589, 2283-2589, 2284-2589, 2285- 2589, 2286-2589, 2287-2589, 2288-2589, 2289-2589, 2290-2589, 2291-2589, 2292-2589, 2293-2589, 2294-2589, 2295-2589, 2296-2589, 2297-2589, 2298-2589, 2299-2589, 2300- 2589, 2301-2589, 2302-2589, 2303-2589, 2304-2589, 2305-2589, 2306-2589, 2307-2589, 2308-2589, 2309-2589, 2310-2589, 2311-2589, 2312-2589, 2313-2589, 2314-2589, 2315- 2589, 2316-2589, 2317-2589, 2318-2589, 2319-2589, 2320-2589, 2321-2589, 2322-2589, 2323-2589, 2324-2589, 2325-2589, 2326-2589, 2327-2589, 2328-2589, 2329-2589, 2330- 2589, 2331-2589, 2332-2589, 2333-2589, 2334-2589, 2335-2589, 2336-2589, 2337-2589, 2338-2589, 2339-2589, 2340-2589, 2341-2589, 2342-2589, 2343-2589, 2344-2589, 2345- 2589, 2346-2589, 2347-2589, 2348-2589, 2349-2589, 2350-2589, 2351-2589, 2352-2589, 2353-2589, 2354-2589, 2355-2589, 2356-2589, 2357-2589, 2358-2589, 2359-2589, 2360- 2589, 2361-2589, 2362-2589, 2363-2589, 2364-2589, 2365-2589, 2366-2589, 2367-2589, 2368-2589, 2369-2589, 2370-2589, 2371-2589, 2372-2589, 2373-2589, 2374-2589, 2375- 2589, 2376-2589, 2377-2589, 2378-2589, 2379-2589, 2380-2589, 2381-2589, 2382-2589, 2383-2589, 2384-2589, 2385-2589, 2386-2589, 2387-2589, 2388-2589, 2389-2589, 2390- 2589, 2391-2589, 2392-2589, 2393-2589, 2394-2589, 2395-2589, 2396-2589, 2397-2589, 2398-2589, 2399-2589, 2400-2589, 2401-2589, 2402-2589, 2403-2589, 2404-2589, 2405- 2589, 2406-2589, 2407-2589, 2408-2589, 2409-2589, 2410-2589, 2411-2589, 2412-2589, 2413-2589, 2414-2589, 2415-2589, 2416-2589, 2417-2589, 2418-2589, 2419-2589, 2420- 2589, 2421-2589, 2422-2589, 2423-2589, 2424-2589, 2425-2589, 2426-2589, 2427-2589, 2428-2589, 2429-2589, 2430-2589, 2431-2589, 2432-2589, 2433-2589, 2434-2589, 2435- 2589, 2436-2589, 2437-2589, 2438-2589, 2439-2589, 2440-2589, 2441-2589, 2442-2589, 2443-2589, 2444-2589, 2445-2589, 2446-2589, 2447-2589, 2448-2589, 2449-2589, 2450- 2589, 2451-2589, 2452-2589, 2453-2589, 2454-2589, 2455-2589, 2456-2589, 2457-2589, 2458-2589, 2459-2589, 2460-2589, 2461-2589, 2462-2589, 2463-2589, 2464-2589, 2465- 2589, 2466-2589, 2467-2589, 2468-2589, 2469-2589, 2470-2589, 2471-2589, 2472-2589, 2473-2589, 2474-2589, 2475-2589, 2476-2589, 2477-2589, 2478-2589, 2479-2589, 2480- 2589, 2481-2589, 2482-2589, 2483-2589, 2484-2589, 2485-2589, 2486-2589, 2487-2589, 2488-2589, 2489-2589, or 2490-2589 of SEQ ID NO: 1, or a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity thereto. A further full-length podocin promoter is shown below as SEQ ID NO: 34. In some embodiments, the full-length podocin promoter sequence has at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 34 shown below. SEQ ID NO: 34 GCCCTCCTATTTAGTCTCTCTGCCACCTACAAATTGAGAAAGTCAAATTTAGTAAGTCCT TATAGTTTCCAGCTCTAAAACAACAGGATTTGTAACATCATGTAGTGCCAATCAATTCCA GCCTTTCCATTAAATAATCCAAATGAATCAGTATTCATGCATTCACTTATTCATATGGATA TAATACTTCAAAACACTAACTGGGAATAAGTGTAATACTTTAGAAAGCCTTGCACTCTTT CATAATCTTTCACCTTGGAAACATAAAAGTAAACAGCTTTAATTGATCCTCCCTTTCTTCT AGTAACCCAGTTCCCACAGATGTCAGTTTCTTAACAAATCCAAACAAGGAGTAAAGTATT GAGAGTTTCAGAATGTGTGCATATCATATTTTCAGCAAAGGGATGAGCAACACTTTCAAT TAAAAAGGAATCTAAAAGAAAACAGCAGCATCCTTGTAAAAGTAGATGAGGACAGCCAG CTGGACTTAGCTGCTAACTCAGCACTCCCTTCCCCAACTCCTCCACCTTCCAGCCATGC TGCATTGACCTTTTTATCATGGAATGGTATGCAGCTTCTCTGATTTTGTCATTGTCACTC ATAATCCCAGCAGACTTGTGTTATAAGCAGCTGCTTATATTGCTGTTATGTATAGATATA GAAATGTAACTTGTCAGTGGTCAAAGAAGCCAAGGAAACTGAGCAAAGGTGACTGAGA ACAAGTGCTCAGCATGGGCTCCAACACCCAGTAGGGCCTTTTCTCATTGATTTTTCTCC TACAAGGAATTTCTCTCTTATTGAAAGACAGTACAGTTTGGGGAATATCATTTGACCAGA TATCCTGCTCCATTCTTGGAATGAAAAACAGGTGCAATTTTAGTATAAGAGTCTAGCTAG GGATGCGGATGAAATTAATTTATAACAGGACCAGGCTGAGGAAAAACACCAATAAATGC TACAAATAGTTTGTCACTTTTTCTAACAGGGAAATCTCTAAAGGATGATAAAGATGCTAG CTGTGTTTAATAGCAAATGTTAATTTGGGGGAATATGAAATAATCATGATATCTTTCTCTC TTTGAGCCAAACGCAGCTTTGGAATTTTTCTCACTGTAGTGTTGTAGGCAGAAACTATCA ACTGAGGCAGGCCCTTGCGAGGAAATATTTATCCTCCTTAGCCCATGCAATGATCAGGT TTAGACAGTAAAAAATCTATTTAAGTGACTGGATTGCTCATCTTTTATTTACTTTACTCCC AGTGGCCTAACCTGGAACATATAGAAGAAAGTAGTGGAATGGTTTTTCCAGACGCACTT CAGTTACTTCAGGTCCTCAGTGTTTAATAAGGTTTTGGGAGAGAAAAAAAGCTATTGCT GTGTGAATTCAATAAATATTTAAAAAATTTAAAAAATCAGTGAGCATTATTTCTGCCATCA TGTGATCTGAACTATCTGAGTAGTATCATAAATCTTGGGTGATGGGTTACCTTGACCAAC CTCCCTTTCAGTAAGAAACAAATTATTAACAGAAAGTGAAAGAGAAATAAACCTATTCAA TGTTTCAAGTCCCTCAGAAGAGGGTGAGGCAGCTCCCCAGATCGTTGCTAGATCCAGC CTGGCCAATGAACCCTGAAAAAGCCCAACTCCTGCTTTCATCATGGAAGCACGGGACA AAGTGTCTCTTCCTAAAAACAGAAGTTAGACCAGACCCCTTCCTGCCTATGATTCTTCAA GAAGCATTGCATCATCAACATCAGGCATAAGCATTAATAAAGACCCTAAATAATAACAGA GACGAAACACATCGCAAAGAGAGTTTTCTTTTATCCCCTTTAAAATGTAAATACTCCCAG GAGGAATCAGCCAACATCATTAGGGGTTAATGCATATGTAGAATAACTAGGGCCAGGAT ATAAAATAAGAAATACGTAGGGAGGAGAGAAAGGCATCCTTGAGACGACTCCAAGAAG GAAAGTTGGGGATGAGGCGAAATTTCTGATTTTACCTTAAAGTGACCCTAATTCGATGA CCTTTTGTGGTTTTTTTCTTTTTTCTTTTTTCTTTTTTACTTGGCCCTGCCCAAGCAGGAC CTAAAAACAAACAGACAAAAAAGGTTACTAACAACTGTTCCTCTCCACGAAAATCTGCAG TAAAAGGTAAAAGATGTATTCGTTTTGAAGAGAAACCAGAGCTTGCGATGAGCTTCTGT ATCTCCGTCAGCCCTCTAGCATGACATTAGGAACCCTCCAGGAGATGAGTCTTCACAGC CCGGGTTGGCACCTGCAGACACGCACTTTTCAACGCCCGCACCCTGCCCGGGGCCGG CTCTCCCACCCAGGCCTCTCTCTGCTTCAGCGCCGCCCCGGCCGTGGGAGTCGGCGG GCGCAGTCCACAGCTCCACCAAGACACAGCTGTCGGGGTTCCGGGTGCGCCCCGCCC GCGGCCCCGGTGTCCCGCCCCTCGCCCTCAGCCCCCACCCGACGGTCTTTAGGGTCC CCCGGGCACGCCACGCGGACCCGCAGCGACTCCACAGGGACTGCGCTCCCGTGCCC CTAGCGCTCCCGCGCTGCTGCTCCAGCCGCCCGGCAGCTCTGACC The 5’-truncated podocin promoter may have a length of about 1.5 kb or less, about 1.4 kb or less, about 1.3 kb or less, about 1.2 kb or less, about 1.1 kb or less, about 1.0 kb or less, about 0.9 kb or less, about 0.8 kb or less, about 0.7 kb or less, or about 0.6 kb or less. In one embodiment the 5’-truncated podocin promoter has a length of 628 bp or less. In one embodiment the 5'-truncated podocin promoter has a length of 578-678 bp, 583-673 bp, 588- 668 bp, 593-663 bp, 598-658 bp, 603-653 bp, 608-648 bp, 613-643 bp, 618-638 bp, or 623- 633 bp. In one embodiment the 5’-truncated podocin promoter has a length of 628 bp. In one embodiment the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 2 shown below. In one embodiment the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 2 shown below. In one embodiment the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least at least 97.0%, at least 97.1%, at least 97.2%, at least 97.3%, at least 97.4%, at least 97.5%, at least 97.6%, at least 97.7%, at least 97.8%, at least 97.9%, at least 98.0%, at least 98.1%, at least 98.2%, at least 98.3%, at least 98.4%, at least 98.5%, at least 98.6%, at least 98.7%, at least 98.8%, at least 98.9%, at least 99.0%, at least 99.1%, at least 99.2%, at least 99.3%, at least 99.4%, at least 99.5%, at least 99.6%, at least 99.7%, at least 99.8%, at least 99.9%, or 100.0% identity to SEQ ID NO: 2. SEQ ID NO: 2. CCAAGAAGGAAAGTTGGGGATGAGGCGAAATTTCTGATTTTACCTTAAAGTGACCCTAA TTCGATGACCTTTTGTGGTTTTTTTCTTTTTTCTTTTTTACTTGGCCCTGCCCAAGCAGGA CCTAAAAACAAACAGACAAAAAAGGTTACTAACAACTGTTCCTCTCCACGAAAATCTGCA GTAAAAGGTAAAAGATGTATTCGTTTTGAAGAGAAACCAGAGCTTGCGATGAGCTTCTG TATCTCCGTCAGCCCTCTAGCATGACATTAGGAACCCTCCAGGAGATGAGTCTTCACAG CCCGGGTTGGCACCTGCAGACACGCACTTTTCAACGCCCGCACCCTGCCCGGGGCCG GCTCTCCCACCCAGGCCTCTCTCTGCTTCAGCGCCGCCCCGGCCGTGGGAGTCGGCG GGCGCAGTCCACAGCTCCACCAAGACACAGCTGTCGGGGTTCCGGGTGCGCCCCGCC CGCGGCCCCGGTGTCCCGCCCCTCGCCCTCAGCCCCCACCCGATGGTCTTTAGGGTC CCCCGGGCACGCCACGCGGACCCGCAGCGACTCCACAGGGACTGCGCTCCCGTGCC CCTAGCGCTCCCGCGCTGCTGCTCCAGCCGCCCGGCAGCTCTGACC A further 5’-truncated podocin promoter is shown below as SEQ ID NO: 35. In one embodiment the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 35 shown below. In one embodiment the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 35 shown below. In one embodiment the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least at least 97.0%, at least 97.1%, at least 97.2%, at least 97.3%, at least 97.4%, at least 97.5%, at least 97.6%, at least 97.7%, at least 97.8%, at least 97.9%, at least 98.0%, at least 98.1%, at least 98.2%, at least 98.3%, at least 98.4%, at least 98.5%, at least 98.6%, at least 98.7%, at least 98.8%, at least 98.9%, at least 99.0%, at least 99.1%, at least 99.2%, at least 99.3%, at least 99.4%, at least 99.5%, at least 99.6%, at least 99.7%, at least 99.8%, at least 99.9%, or 100.0% identity to SEQ ID NO: 35. SEQ ID NO: 35 GGAAAGTTGGGGATGAGGCGAAATTTCTGATTTTACCTTAAAGTGACCCTAATTCGATG ACCTTTTGTGGTTTTTTTCTTTTTTCTTTTTTCTTTTTTACTTGGCCCTGCCCAAGCAGGA CCTAAAAACAAACAGACAAAAAAGGTTACTAACAACTGTTCCTCTCCACGAAAATCTGCA GTAAAAGGTAAAAGATGTATTCGTTTTGAAGAGAAACCAGAGCTTGCGATGAGCTTCTG TATCTCCGTCAGCCCTCTAGCATGACATTAGGAACCCTCCAGGAGATGAGTCTTCACAG CCCGGGTTGGCACCTGCAGACACGCACTTTTCAACGCCCGCACCCTGCCCGGGGCCG GCTCTCCCACCCAGGCCTCTCTCTGCTTCAGCGCCGCCCCGGCCGTGGGAGTCGGCG GGCGCAGTCCACAGCTCCACCAAGACACAGCTGTCGGGGTTCCGGGTGCGCCCCGCC CGCGGCCCCGGTGTCCCGCCCCTCGCCCTCAGCCCCCACCCGACGGTCTTTAGGGTC CCCCGGGCACGCCACGCGGACCCGCAGCGACTCCACAGGGACTGCGCTCCCGTGCC CCTAGCGCTCCCGCGCTGCTGCTCCAGCCGCCCGGCAGCTCTGACC In one embodiment the 5’-truncated podocin promoter has a length of 600 bp or less. In one embodiment the 5’-truncated podocin promoter has a length of 550-650 bp, 555-645 bp, 560- 640 bp, 565-635 bp, 570-630 bp, 575-625 bp, 580-620 bp, 585-615 bp, 590-610 bp, or 595- 605 bp. In one embodiment the 5’-truncated podocin promoter has a length of 600 bp. In one embodiment the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 36 shown below. In one embodiment the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 36 shown below. SEQ ID NO: 36 AATTTCTGATTTTACCTTAAAGTGACCCTAATTCGATGACCTTTTGTGGTTTTTTTCTTTTT TCTTTTTTACTTGGCCCTGCCCAAGCAGGACCTAAAAACAAACAGACAAAAAAGGTTAC TAACAACTGTTCCTCTCCACGAAAATCTGCAGTAAAAGGTAAAAGATGTATTCGTTTTGA AGAGAAACCAGAGCTTGCGATGAGCTTCTGTATCTCCGTCAGCCCTCTAGCATGACATT AGGAACCCTCCAGGAGATGAGTCTTCACAGCCCGGGTTGGCACCTGCAGACACGCACT TTTCAACGCCCGCACCCTGCCCGGGGCCGGCTCTCCCACCCAGGCCTCTCTCTGCTTC AGCGCCGCCCCGGCCGTGGGAGTCGGCGGGCGCAGTCCACAGCTCCACCAAGACAC AGCTGTCGGGGTTCCGGGTGCGCCCCGCCCGCGGCCCCGGTGTCCCGCCCCTCGCC CTCAGCCCCCACCCGATGGTCTTTAGGGTCCCCCGGGCACGCCACGCGGACCCGCAG CGACTCCACAGGGACTGCGCTCCCGTGCCCCTAGCGCTCCCGCGCTGCTGCTCCAGC CGCCCGGCAGCTCTGACC In one embodiment the 5’-truncated podocin promoter has a length of 578 bp or less. In one embodiment the 5’-truncated podocin promoter has a length of 528-628 bp, 533-623 bp, 538- 618 bp, 543-613 bp, 548-608 bp, 553-603 bp, 558-598 bp, 563-593 bp, 568-588 bp, or 573- 583 bp. In one embodiment the 5’-truncated podocin promoter has a length of 578 bp. In one embodiment the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 47 shown below. In one embodiment the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 47 shown below. SEQ ID NO: 47 TGACCCTAATTCGATGACCTTTTGTGGTTTTTTTCTTTTTTCTTTTTTACTTGGCCCTGCC CAAGCAGGACCTAAAAACAAACAGACAAAAAAGGTTACTAACAACTGTTCCTCTCCACG AAAATCTGCAGTAAAAGGTAAAAGATGTATTCGTTTTGAAGAGAAACCAGAGCTTGCGA TGAGCTTCTGTATCTCCGTCAGCCCTCTAGCATGACATTAGGAACCCTCCAGGAGATGA GTCTTCACAGCCCGGGTTGGCACCTGCAGACACGCACTTTTCAACGCCCGCACCCTGC CCGGGGCCGGCTCTCCCACCCAGGCCTCTCTCTGCTTCAGCGCCGCCCCGGCCGTGG GAGTCGGCGGGCGCAGTCCACAGCTCCACCAAGACACAGCTGTCGGGGTTCCGGGTG CGCCCCGCCCGCGGCCCCGGTGTCCCGCCCCTCGCCCTCAGCCCCCACCCGATGGT CTTTAGGGTCCCCCGGGCACGCCACGCGGACCCGCAGCGACTCCACAGGGACTGCGC TCCCGTGCCCCTAGCGCTCCCGCGCTGCTGCTCCAGCCGCCCGGCAGCTCTGACC In one embodiment the 5’-truncated podocin promoter has a length of 550 bp or less. In one embodiment the 5’-truncated podocin promoter has a length of 500-600 bp, 505-595 bp, 510- 590 bp, 515-585 bp, 520-580 bp, 525-575 bp, 530-570 bp, 535-565 bp, 540-560 bp, or 545- 555 bp. In one embodiment the 5’-truncated podocin promoter has a length of 550 bp. In one embodiment the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 37 shown below. In one embodiment the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 37 shown below. SEQ ID NO: 37 TTTTTTCTTTTTTCTTTTTTACTTGGCCCTGCCCAAGCAGGACCTAAAAACAAACAGACA AAAAAGGTTACTAACAACTGTTCCTCTCCACGAAAATCTGCAGTAAAAGGTAAAAGATGT ATTCGTTTTGAAGAGAAACCAGAGCTTGCGATGAGCTTCTGTATCTCCGTCAGCCCTCT AGCATGACATTAGGAACCCTCCAGGAGATGAGTCTTCACAGCCCGGGTTGGCACCTGC AGACACGCACTTTTCAACGCCCGCACCCTGCCCGGGGCCGGCTCTCCCACCCAGGCC TCTCTCTGCTTCAGCGCCGCCCCGGCCGTGGGAGTCGGCGGGCGCAGTCCACAGCTC CACCAAGACACAGCTGTCGGGGTTCCGGGTGCGCCCCGCCCGCGGCCCCGGTGTCC CGCCCCTCGCCCTCAGCCCCCACCCGATGGTCTTTAGGGTCCCCCGGGCACGCCACG CGGACCCGCAGCGACTCCACAGGGACTGCGCTCCCGTGCCCCTAGCGCTCCCGCGCT GCTGCTCCAGCCGCCCGGCAGCTCTGACC In one embodiment the 5’-truncated podocin promoter has a length of 528 bp or less. In one embodiment the 5'-truncated podocin promoter has a length of 478-578 bp, 483-573 bp, 488- 568 bp, 493-563 bp, 498-558 bp, 503-553 bp, 508-548 bp, 513-543 bp, 518-538 bp, or 523- 533 bp. In one embodiment the 5’-truncated podocin promoter has a length of 528 bp. In one embodiment the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 48 shown below. In one embodiment the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 48 shown below. SEQ ID NO: 48 TTGGCCCTGCCCAAGCAGGACCTAAAAACAAACAGACAAAAAAGGTTACTAACAACTGT TCCTCTCCACGAAAATCTGCAGTAAAAGGTAAAAGATGTATTCGTTTTGAAGAGAAACCA GAGCTTGCGATGAGCTTCTGTATCTCCGTCAGCCCTCTAGCATGACATTAGGAACCCTC CAGGAGATGAGTCTTCACAGCCCGGGTTGGCACCTGCAGACACGCACTTTTCAACGCC CGCACCCTGCCCGGGGCCGGCTCTCCCACCCAGGCCTCTCTCTGCTTCAGCGCCGCC CCGGCCGTGGGAGTCGGCGGGCGCAGTCCACAGCTCCACCAAGACACAGCTGTCGG GGTTCCGGGTGCGCCCCGCCCGCGGCCCCGGTGTCCCGCCCCTCGCCCTCAGCCCC CACCCGATGGTCTTTAGGGTCCCCCGGGCACGCCACGCGGACCCGCAGCGACTCCAC AGGGACTGCGCTCCCGTGCCCCTAGCGCTCCCGCGCTGCTGCTCCAGCCGCCCGGCA GCTCTGACC In one embodiment the 5’-truncated podocin promoter has a length of 500 bp or less. In one embodiment the 5'-truncated podocin promoter has a length of 450-550 bp, 455-545 bp, 460- 540 bp, 465-535 bp, 470-530 bp, 475-525 bp, 480-520 bp, 485-515 bp, 490-510 bp, or 495- 505 bp. In one embodiment the 5’-truncated podocin promoter has a length of 500 bp. In one embodiment the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 38 shown below. In one embodiment the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 38 shown below. SEQ ID NO: 38 CAAACAGACAAAAAAGGTTACTAACAACTGTTCCTCTCCACGAAAATCTGCAGTAAAAG GTAAAAGATGTATTCGTTTTGAAGAGAAACCAGAGCTTGCGATGAGCTTCTGTATCTCC GTCAGCCCTCTAGCATGACATTAGGAACCCTCCAGGAGATGAGTCTTCACAGCCCGGG TTGGCACCTGCAGACACGCACTTTTCAACGCCCGCACCCTGCCCGGGGCCGGCTCTC CCACCCAGGCCTCTCTCTGCTTCAGCGCCGCCCCGGCCGTGGGAGTCGGCGGGCGC AGTCCACAGCTCCACCAAGACACAGCTGTCGGGGTTCCGGGTGCGCCCCGCCCGCGG CCCCGGTGTCCCGCCCCTCGCCCTCAGCCCCCACCCGATGGTCTTTAGGGTCCCCCG GGCACGCCACGCGGACCCGCAGCGACTCCACAGGGACTGCGCTCCCGTGCCCCTAG CGCTCCCGCGCTGCTGCTCCAGCCGCCCGGCAGCTCTGACC In one embodiment the 5’-truncated podocin promoter has a length of 478 bp or less. In one embodiment the 5'-truncated podocin promoter has a length of 428-528 bp, 433-523 bp, 438- 518 bp, 443-513 bp, 448-508 bp, 453-503 bp, 458-498 bp, 463-493 bp, 468-488 bp, or 473- 483 bp. In one embodiment the 5’-truncated podocin promoter has a length of 478 bp. In one embodiment the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 49 shown below. In one embodiment the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 49 shown below. SEQ ID NO: 49 AACAACTGTTCCTCTCCACGAAAATCTGCAGTAAAAGGTAAAAGATGTATTCGTTTTGAA GAGAAACCAGAGCTTGCGATGAGCTTCTGTATCTCCGTCAGCCCTCTAGCATGACATTA GGAACCCTCCAGGAGATGAGTCTTCACAGCCCGGGTTGGCACCTGCAGACACGCACTT TTCAACGCCCGCACCCTGCCCGGGGCCGGCTCTCCCACCCAGGCCTCTCTCTGCTTCA GCGCCGCCCCGGCCGTGGGAGTCGGCGGGCGCAGTCCACAGCTCCACCAAGACACA GCTGTCGGGGTTCCGGGTGCGCCCCGCCCGCGGCCCCGGTGTCCCGCCCCTCGCCC TCAGCCCCCACCCGATGGTCTTTAGGGTCCCCCGGGCACGCCACGCGGACCCGCAGC GACTCCACAGGGACTGCGCTCCCGTGCCCCTAGCGCTCCCGCGCTGCTGCTCCAGCC GCCCGGCAGCTCTGACC In one embodiment the 5’-truncated podocin promoter has a length of 450 bp or less. In one embodiment the 5'-truncated podocin promoter has a length of 400-500 bp, 405-495 bp, 410- 490 bp, 415-485 bp, 420-480 bp, 425-475 bp, 430-470 bp, 435-465 bp, 440-460 bp, or 445- 455 bp. In one embodiment the 5’-truncated podocin promoter has a length of 450 bp. In one embodiment the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 39 shown below. In one embodiment the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 39 shown below. SEQ ID NO: 39 CAGTAAAAGGTAAAAGATGTATTCGTTTTGAAGAGAAACCAGAGCTTGCGATGAGCTTC TGTATCTCCGTCAGCCCTCTAGCATGACATTAGGAACCCTCCAGGAGATGAGTCTTCAC AGCCCGGGTTGGCACCTGCAGACACGCACTTTTCAACGCCCGCACCCTGCCCGGGGC CGGCTCTCCCACCCAGGCCTCTCTCTGCTTCAGCGCCGCCCCGGCCGTGGGAGTCGG CGGGCGCAGTCCACAGCTCCACCAAGACACAGCTGTCGGGGTTCCGGGTGCGCCCCG CCCGCGGCCCCGGTGTCCCGCCCCTCGCCCTCAGCCCCCACCCGATGGTCTTTAGGG TCCCCCGGGCACGCCACGCGGACCCGCAGCGACTCCACAGGGACTGCGCTCCCGTG CCCCTAGCGCTCCCGCGCTGCTGCTCCAGCCGCCCGGCAGCTCTGACC In one embodiment the 5’-truncated podocin promoter has a length of 428 bp or less. In one embodiment the 5'-truncated podocin promoter has a length of 378-478 bp, 383-473 bp, 388- 468 bp, 393-463 bp, 398-458 bp, 403-453 bp, 408-448 bp, 413-443 bp, 418-438 bp, or 423- 433 bp. In one embodiment the 5’-truncated podocin promoter has a length of 428 bp. In one embodiment the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 50 shown below. In one embodiment the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 50 shown below. SEQ ID NO: 50 TCGTTTTGAAGAGAAACCAGAGCTTGCGATGAGCTTCTGTATCTCCGTCAGCCCTCTAG CATGACATTAGGAACCCTCCAGGAGATGAGTCTTCACAGCCCGGGTTGGCACCTGCAG ACACGCACTTTTCAACGCCCGCACCCTGCCCGGGGCCGGCTCTCCCACCCAGGCCTC TCTCTGCTTCAGCGCCGCCCCGGCCGTGGGAGTCGGCGGGCGCAGTCCACAGCTCCA CCAAGACACAGCTGTCGGGGTTCCGGGTGCGCCCCGCCCGCGGCCCCGGTGTCCCG CCCCTCGCCCTCAGCCCCCACCCGATGGTCTTTAGGGTCCCCCGGGCACGCCACGCG GACCCGCAGCGACTCCACAGGGACTGCGCTCCCGTGCCCCTAGCGCTCCCGCGCTGC TGCTCCAGCCGCCCGGCAGCTCTGACC In one embodiment the 5’-truncated podocin promoter has a length of 400 bp or less. In one embodiment the 5'-truncated podocin promoter has a length of 350-450 bp, 355-445 bp, 360- 440 bp, 365-435 bp, 370-430 bp, 375-425 bp, 380-420 bp, 385-415 bp, 390-410 bp, or 395- 405 bp. In one embodiment the 5’-truncated podocin promoter has a length of 400 bp. In one embodiment the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 40 shown below. In one embodiment the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 40 shown below. SEQ ID NO: 40 ATGAGCTTCTGTATCTCCGTCAGCCCTCTAGCATGACATTAGGAACCCTCCAGGAGATG AGTCTTCACAGCCCGGGTTGGCACCTGCAGACACGCACTTTTCAACGCCCGCACCCTG CCCGGGGCCGGCTCTCCCACCCAGGCCTCTCTCTGCTTCAGCGCCGCCCCGGCCGTG GGAGTCGGCGGGCGCAGTCCACAGCTCCACCAAGACACAGCTGTCGGGGTTCCGGGT GCGCCCCGCCCGCGGCCCCGGTGTCCCGCCCCTCGCCCTCAGCCCCCACCCGATGG TCTTTAGGGTCCCCCGGGCACGCCACGCGGACCCGCAGCGACTCCACAGGGACTGCG CTCCCGTGCCCCTAGCGCTCCCGCGCTGCTGCTCCAGCCGCCCGGCAGCTCTGACC In one embodiment the 5’-truncated podocin promoter has a length of 378 bp or less. In one embodiment the 5'-truncated podocin promoter has a length of 328-428 bp, 333-423 bp, 338- 418 bp, 343-413 bp, 348-408 bp, 353-403 bp, 358-398 bp, 363-393 bp, 368-388 bp, or 373- 383 bp. In one embodiment the 5’-truncated podocin promoter has a length of 378 bp. In one embodiment the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 51 shown below. In one embodiment the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 51 shown below. SEQ ID NO: 51 GCCCTCTAGCATGACATTAGGAACCCTCCAGGAGATGAGTCTTCACAGCCCGGGTTGG CACCTGCAGACACGCACTTTTCAACGCCCGCACCCTGCCCGGGGCCGGCTCTCCCAC CCAGGCCTCTCTCTGCTTCAGCGCCGCCCCGGCCGTGGGAGTCGGCGGGCGCAGTC CACAGCTCCACCAAGACACAGCTGTCGGGGTTCCGGGTGCGCCCCGCCCGCGGCCCC GGTGTCCCGCCCCTCGCCCTCAGCCCCCACCCGATGGTCTTTAGGGTCCCCCGGGCA CGCCACGCGGACCCGCAGCGACTCCACAGGGACTGCGCTCCCGTGCCCCTAGCGCTC CCGCGCTGCTGCTCCAGCCGCCCGGCAGCTCTGACC In one embodiment the 5’-truncated podocin promoter has a length of 350 bp or less. In one embodiment the 5'-truncated podocin promoter has a length of 300-400 bp, 305-395 bp, 310- 390 bp, 315-385 bp, 320-380 bp, 325-375 bp, 330-370 bp, 335-365 bp, 340-360 bp, or 345- 355 bp. In one embodiment the 5’-truncated podocin promoter has a length of 350 bp. In one embodiment the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 41 shown below. In one embodiment the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 41 shown below. SEQ ID NO: 41 CAGGAGATGAGTCTTCACAGCCCGGGTTGGCACCTGCAGACACGCACTTTTCAACGCC CGCACCCTGCCCGGGGCCGGCTCTCCCACCCAGGCCTCTCTCTGCTTCAGCGCCGCC CCGGCCGTGGGAGTCGGCGGGCGCAGTCCACAGCTCCACCAAGACACAGCTGTCGG GGTTCCGGGTGCGCCCCGCCCGCGGCCCCGGTGTCCCGCCCCTCGCCCTCAGCCCC CACCCGATGGTCTTTAGGGTCCCCCGGGCACGCCACGCGGACCCGCAGCGACTCCAC AGGGACTGCGCTCCCGTGCCCCTAGCGCTCCCGCGCTGCTGCTCCAGCCGCCCGGCA GCTCTGACC In one embodiment the 5’-truncated podocin promoter has a length of 328 bp or less. In one embodiment the 5'-truncated podocin promoter has a length of 278-378 bp, 283-373 bp, 288- 368 bp, 293-363 bp, 298-358 bp, 303-353 bp, 308-348 bp, 313-343 bp, 318-338 bp, or 323- 333 bp. In one embodiment the 5’-truncated podocin promoter has a length of 328 bp. In one embodiment the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 52 shown below. In one embodiment the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 52 shown below. SEQ ID NO: 52 CGGGTTGGCACCTGCAGACACGCACTTTTCAACGCCCGCACCCTGCCCGGGGCCGGC TCTCCCACCCAGGCCTCTCTCTGCTTCAGCGCCGCCCCGGCCGTGGGAGTCGGCGGG CGCAGTCCACAGCTCCACCAAGACACAGCTGTCGGGGTTCCGGGTGCGCCCCGCCCG CGGCCCCGGTGTCCCGCCCCTCGCCCTCAGCCCCCACCCGATGGTCTTTAGGGTCCC CCGGGCACGCCACGCGGACCCGCAGCGACTCCACAGGGACTGCGCTCCCGTGCCCC TAGCGCTCCCGCGCTGCTGCTCCAGCCGCCCGGCAGCTCTGACC In one embodiment the 5’-truncated podocin promoter has a length of 300 bp or less. In one embodiment the 5'-truncated podocin promoter has a length of 250-350 bp, 255-345 bp, 260- 340 bp, 265-335 bp, 270-330 bp, 275-325 bp, 280-320 bp, 285-315 bp, 290-310 bp, or 295- 305 bp. In one embodiment the 5’-truncated podocin promoter has a length of 300 bp. In one embodiment the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 42 shown below. In one embodiment the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 42 shown below. SEQ ID NO: 42 TCAACGCCCGCACCCTGCCCGGGGCCGGCTCTCCCACCCAGGCCTCTCTCTGCTTCA GCGCCGCCCCGGCCGTGGGAGTCGGCGGGCGCAGTCCACAGCTCCACCAAGACACA GCTGTCGGGGTTCCGGGTGCGCCCCGCCCGCGGCCCCGGTGTCCCGCCCCTCGCCC TCAGCCCCCACCCGATGGTCTTTAGGGTCCCCCGGGCACGCCACGCGGACCCGCAGC GACTCCACAGGGACTGCGCTCCCGTGCCCCTAGCGCTCCCGCGCTGCTGCTCCAGCC GCCCGGCAGCTCTGACC In one embodiment the 5’-truncated podocin promoter has a length of 278 bp or less. In one embodiment the 5'-truncated podocin promoter has a length of 228-328 bp, 233-323 bp, 238- 318 bp, 243-313 bp, 248-308 bp, 253-303 bp, 258-298 bp, 263-293 bp, 268-288 bp, or 273- 283 bp. In one embodiment the 5’-truncated podocin promoter has a length of 278 bp. In one embodiment the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 53 shown below. In one embodiment the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 53 shown below. SEQ ID NO: 53 GGCCGGCTCTCCCACCCAGGCCTCTCTCTGCTTCAGCGCCGCCCCGGCCGTGGGAGT CGGCGGGCGCAGTCCACAGCTCCACCAAGACACAGCTGTCGGGGTTCCGGGTGCGCC CCGCCCGCGGCCCCGGTGTCCCGCCCCTCGCCCTCAGCCCCCACCCGATGGTCTTTA GGGTCCCCCGGGCACGCCACGCGGACCCGCAGCGACTCCACAGGGACTGCGCTCCC GTGCCCCTAGCGCTCCCGCGCTGCTGCTCCAGCCGCCCGGCAGCTCTGACC In one embodiment the 5’-truncated podocin promoter has a length of 250 bp or less. In one embodiment the 5'-truncated podocin promoter has a length of 200-300 bp, 205-295 bp, 210- 290 bp, 215-285 bp, 220-280 bp, 225-275 bp, 230-270 bp, 235-265 bp, 240-260 bp, or 245- 255 bp. In one embodiment the 5’-truncated podocin promoter has a length of 250 bp. In one embodiment the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 43 shown below. In one embodiment the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 43 shown below. SEQ ID NO: 43 TGCTTCAGCGCCGCCCCGGCCGTGGGAGTCGGCGGGCGCAGTCCACAGCTCCACCAA GACACAGCTGTCGGGGTTCCGGGTGCGCCCCGCCCGCGGCCCCGGTGTCCCGCCCC TCGCCCTCAGCCCCCACCCGATGGTCTTTAGGGTCCCCCGGGCACGCCACGCGGACC CGCAGCGACTCCACAGGGACTGCGCTCCCGTGCCCCTAGCGCTCCCGCGCTGCTGCT CCAGCCGCCCGGCAGCTCTGACC In one embodiment the 5’-truncated podocin promoter has a length of 228 bp or less. In one embodiment the 5'-truncated podocin promoter has a length of 178-278 bp, 183-273 bp, 188- 268 bp, 193-263 bp, 198-258 bp, 203-253 bp, 208-248 bp, 213-243 bp, 218-238 bp, or 223- 233 bp. In one embodiment the 5’-truncated podocin promoter has a length of 228 bp. In one embodiment the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 54 shown below. In one embodiment the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 54 shown below. SEQ ID NO: 54 TGGGAGTCGGCGGGCGCAGTCCACAGCTCCACCAAGACACAGCTGTCGGGGTTCCGG GTGCGCCCCGCCCGCGGCCCCGGTGTCCCGCCCCTCGCCCTCAGCCCCCACCCGAT GGTCTTTAGGGTCCCCCGGGCACGCCACGCGGACCCGCAGCGACTCCACAGGGACTG CGCTCCCGTGCCCCTAGCGCTCCCGCGCTGCTGCTCCAGCCGCCCGGCAGCTCTGAC C In one embodiment the 5’-truncated podocin promoter has a length of 200 bp or less. In one embodiment the 5'-truncated podocin promoter has a length of 150-250 bp, 155-245 bp, 160- 240 bp, 165-235 bp, 170-230 bp, 175-225 bp, 180-220 bp, 185-215 bp, 190-210 bp, or 195- 205 bp. In one embodiment the 5’-truncated podocin promoter has a length of 200 bp. In one embodiment the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 44 shown below. In one embodiment the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 44 shown below. SEQ ID NO: 44 CCACCAAGACACAGCTGTCGGGGTTCCGGGTGCGCCCCGCCCGCGGCCCCGGTGTC CCGCCCCTCGCCCTCAGCCCCCACCCGATGGTCTTTAGGGTCCCCCGGGCACGCCAC GCGGACCCGCAGCGACTCCACAGGGACTGCGCTCCCGTGCCCCTAGCGCTCCCGCG CTGCTGCTCCAGCCGCCCGGCAGCTCTGACC In one embodiment the 5’-truncated podocin promoter has a length of 150 bp or less. In one embodiment the 5'-truncated podocin promoter has a length of 100-200 bp, 105-195 bp, 110- 190 bp, 115-185 bp, 120-180 bp, 125-175 bp, 130-170 bp, 135-165 bp, 140-160 bp, or 145- 155 bp. In one embodiment the 5’-truncated podocin promoter has a length of 150 bp. In one embodiment the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 45 shown below. In one embodiment the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 45 shown below. SEQ ID NO: 45 GGTGTCCCGCCCCTCGCCCTCAGCCCCCACCCGATGGTCTTTAGGGTCCCCCGGGCA CGCCACGCGGACCCGCAGCGACTCCACAGGGACTGCGCTCCCGTGCCCCTAGCGCTC CCGCGCTGCTGCTCCAGCCGCCCGGCAGCTCTGACC In one embodiment the 5’-truncated podocin promoter has a length of 100 bp or less. In one embodiment the 5’-truncated podocin promoter has a length of 100 bp. In one embodiment the 5’-truncated podocin promoter consists or comprises of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 46 shown below. In one embodiment the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 46 shown below. SEQ ID NO: 46 CCGGGCACGCCACGCGGACCCGCAGCGACTCCACAGGGACTGCGCTCCCGTGCCCC TAGCGCTCCCGCGCTGCTGCTCCAGCCGCCCGGCAGCTCTGACC The 5’-truncated podocin promoter may have a length of about 100 bp or more, 150 bp or more, 200 bp or more, 250 bp or more, 300 bp or more, 350 bp or more, 400 bp or more, 450 bp or more, 500 bp or more, 550 bp or more, or 600 bp or more. In one embodiment, the 5’- truncated podocin promoter has a length of about 150 bp or more. In one embodiment, the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to any of SEQ ID NOs: 2 or 35-54. In one embodiment, the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to any of SEQ ID NOs: 36-54. In one embodiment, the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to any of SEQ ID NOs: 36-46. In one embodiment, the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to any of SEQ ID NOs: 47-54. In one embodiment, the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to any of SEQ ID NOs: 38, 40, 41, 42, 44, 47, 48, 51, 53. In one embodiment, the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to any of SEQ ID NOs: 40, 47, 53. Polynucleotides In one aspect, the present invention provides a polynucleotide comprising the promoter described herein. Polynucleotides of the invention may comprise DNA or RNA, preferably DNA. They may be single-stranded or double-stranded. Polynucleotides such as DNA polynucleotides may be produced recombinant, synthetically or by any means available to those of skill in the art. They may also be cloned by standard techniques. The polynucleotide may be an isolated polynucleotide. Longer polynucleotides will generally be produced using recombinant means, for example using polymerase chain reaction (PCR) cloning techniques. This will involve making a pair of primers (e.g. of about 15 to 30 nucleotides) flanking the target sequence which it is desired to clone, bringing the primers into contact with mRNA or cDNA obtained from an animal or human cell, performing a polymerase chain reaction under conditions which bring about amplification of the desired region, isolating the amplified fragment (e.g. by purifying the reaction mixture with an agarose gel) and recovering the amplified DNA. The primers may be designed to contain suitable restriction enzyme recognition sites so that the amplified DNA can be cloned into a suitable vector. The polynucleotides may be modified by any method available in the art. Such modifications may be carried out in order to enhance the in vivo activity or lifespan of the polynucleotides of the invention. Transgenes The promoter referred to herein may be operably linked to one or more transgenes. Preferably, the transgene is a therapeutic transgene. The transgene may encode any polypeptide of interest. For example, the transgene sequence man encode any polypeptide associated with a kidney disease such as a glomerular disease. The transgene may encode a polypeptide involved in a GBM-associated genetic glomerular disease. The transgene may encode a polypeptide involved in a podocyte-associated genetic glomerular disease. Suitably, the transgene may encode a COL4A3, COL4A4, COL4A5, NPHS2, CFH, CFL, CFHL1, C1INH, C4BP, MASP2, C3, C5aR1, C5, C5a, CD55, CD35, CD46, CD59, vitronectin, clusterin, ADCK4, ALG1, ARHGAP24, ARGHDIA, CD151, CD2AP, COQ2, COQ6, DGKE, E2F3, EMP2, KANK2, LAGE3, LMNA, LMX1B, MAF B, NUP85, NUP93, NXF5, OSGEP, PAX2, PDSS2, PMM2, PODXL, SCARB2, SGPL1, Smad7, TP53RK, TPRKB, VDR, WDR73, WT1, ZMPSTE24, APOL1, NPHS1, TRPC6, NUP107, NUP133, NUP160, ACTN4, INF2, ANKFY1, ANLN, CRB2, ITGA3, KANK1, KANK4, MAGI2, MYO1E, OCRL, PTPRO, SMARCAL1, SYNPO, TBC1D8B, XPO5, TNS2, NLRP3, or VEGFC polypeptide. For a protein-coding polynucleotide, it will be understood by a skilled person that numerous different polynucleotides can encode the same polypeptide as a result of the degeneracy of the genetic code. In addition, it is to be understood that skilled persons may, using routine techniques, make nucleotide substitutions that do not affect the polypeptide sequence encoded by the polynucleotides of the invention to reflect the codon usage of any particular host organism in which the polypeptides of the invention are to be expressed. The transgene may be codon-optimised. Different cells differ in their usage of particular codons. This codon bias corresponds to a bias in the relative abundance of particular tRNAs in the cell type. By altering the codons in the sequence so that they are tailored to match with the relative abundance of corresponding tRNAs, it is possible to increase expression. Similarly, it is possible to decrease expression by deliberately choosing codons for which the corresponding tRNAs are known to be rare in the particular cell type. Thus, an additional degree of translational control is available. Codon usage tables are known in the art for mammalian cells (e.g. humans), as well as for a variety of other organisms. The transgenes disclosed herein may comprise or lack stop codons at their 3’ end. Thus, the present disclosure encompasses the SEQ ID NOs disclosed herein with the stop codons present or absent. COL4A3, COL4A4 and COL4A5 polypeptides The transgene may encode a COL4A3, COL4A4 or COL4A5 polypeptide, or a fragment or derivative thereof. COL4A3, COL4A4 and COL4A5 proteins are approximately 170-185 kDa homologous polypeptides containing collagenous Gly-X-Y repeat sequences frequently interrupted by non- collagenous sequences and forming a triple helix repeat. Each polypeptide also contains a large globular non-collagenous domain at the carboxyl-terminal end. Alport syndrome (AS) is caused by pathogenic variants in the COL4A3, COL4A4 and COL4A5 genes, which result in abnormalities of the collagen IV α345 network of basement membranes. The COL4A3, COL4A4 or COL4A5 polypeptide or a fragment or derivative thereof may be capable of forming a collagen IV α345 network. Approximately 200-300 amino acids may be removed from each of the COL4A3, COL4A4 and COL4A5 polypeptides to produce a truncated transgene suitable for a mini-gene approach. The amino acids may be removed from the triple helix repeat. Preferably, the amino acids are not removed from the non-collagenous region. In some embodiments, the COL4A3, COL4A4 and COL4A5 polypeptides are full-length polypeptides. Preferably, the COL4A3, COL4A4 or COL4A5 polypeptide is human. An example human COL4A3 is the COL4A3 having the UniProtKB accession number Q01955. An example human COL4A4 is the COL4A4 having the UniProtKB accession number P53420. An example human COL4A5 is the COL4A5 having the UniProtKB accession number P29400. Suitably, the COL4A3 peptide may comprise or consist of the polypeptide sequence shown as SEQ ID NO: 3, or a variant which is at least 70% identical to SEQ ID NO: 3. Suitably, the variant may be at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99% identical to SEQ ID NO: 3. Suitably, the COL4A4 peptide may comprise or consist of the polypeptide sequence shown as SEQ ID NO: 4, or a variant which is at least 70% identical to SEQ ID NO: 4. Suitably, the variant may be at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99% identical to SEQ ID NO: 4. Suitably, the COL4A5 peptide may comprise or consist of the polypeptide sequence shown as SEQ ID NO: 5, or a variant which is at least 70% identical to SEQ ID NO: 5. Suitably, the variant may be at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99% identical to SEQ ID NO: 5. Exemplary COL4A3 amino acid sequence – Uniprot reference Q01955 (SEQ ID NO: 3) MSARTAPRPQVLLLPLLLVLLAAAPAASKGCVCKDKGQCFCDGAKGEKGEKGFPGPPGSPGQKGFTGP EGLPGPQGPKGFPGLPGLTGSKGVRGISGLPGFSGSPGLPGTPGNTGPYGLVGVPGCSGSKGEQGFPG LPGTLGYPGIPGAAGLKGQKGAPAKEEDIELDAKGDPGLPGAPGPQGLPGPPGFPGPVGPPGPPGFFG FPGAMGPRGPKGHMGERVIGHKGERGVKGLTGPPGPPGTVIVTLTGPDNRTDLKGEKGDKGAMGEPGP PGPSGLPGESYGSEKGAPGDPGLQGKPGKDGVPGFPGSEGVKGNRGFPGLMGEDGIKGQKGDIGPPGF RGPTEYYDTYQEKGDEGTPGPPGPRGARGPQGPSGPPGVPGSPGSSRPGLRGAPGWPGLKGSKGERGR PGKDAMGTPGSPGCAGSPGLPGSPGPPGPPGDIVFRKGPPGDHGLPGYLGSPGIPGVDGPKGEPGLLC TQCPYIPGPPGLPGLPGLHGVKGIPGRQGAAGLKGSPGSPGNTGLPGFPGFPGAQGDPGLKGEKGETL QPEGQVGVPGDPGLRGQPGRKGLDGIPGTPGVKGLPGPKGELALSGEKGDQGPPGDPGSPGSPGPAGP AGPPGYGPQGEPGLQGTQGVPGAPGPPGEAGPRGELSVSTPVPGPPGPPGPPGHPGPQGPPGIPGSLG KCGDPGLPGPDGEPGIPGIGFPGPPGPKGDQGFPGTKGSLGCPGKMGEPGLPGKPGLPGAKGEPAVAM PGGPGTPGFPGERGNSGEHGEIGLPGLPGLPGTPGNEGLDGPRGDPGQPGPPGEQGPPGRCIEGPRGA QGLPGLNGLKGQQGRRGKTGPKGDPGIPGLDRSGFPGETGSPGIPGHQGEMGPLGQRGYPGNPGILGP PGEDGVIGMMGFPGAIGPPGPPGNPGTPGQRGSPGIPGVKGQRGTPGAKGEQGDKGNPGPSEISHVIG DKGEPGLKGFAGNPGEKGNRGVPGMPGLKGLKGLPGPAGPPGPRGDLGSTGNPGEPGLRGIPGSMGNM GMPGSKGKRGTLGFPGRAGRPGLPGIHGLQGDKGEPGYSEGTRPGPPGPTGDPGLPGDMGKKGEMGQP GPPGHLGPAGPEGAPGSPGSPGLPGKPGPHGDLGFKGIKGLLGPPGIRGPPGLPGFPGSPGPMGIRGD QGRDGIPGPAGEKGETGLLRAPPGPRGNPGAQGAKGDRGAPGFPGLPGRKGAMGDAGPRGPTGIEGFP GPPGLPGAIIPGQTGNRGPPGSRGSPGAPGPPGPPGSHVIGIKGDKGSMGHPGPKGPPGTAGDMGPPG RLGAPGTPGLPGPRGDPGFQGFPGVKGEKGNPGFLGSIGPPGPIGPKGPPGVRGDPGTLKIISLPGSP GPPGTPGEPGMQGEPGPPGPPGNLGPCGPRGKPGKDGKPGTPGPAGEKGNKGSKGEPGPAGSDGLPGL KGKRGDSGSPATWTTRGFVFTRHSQTTAIPSCPEGTVPLYSGFSFLFVQGNQRAHGQDLGTLGSCLQR FTTMPFLFCNVNDVCNFASRNDYSYWLSTPALMPMNMAPITGRALEPYISRCTVCEGPAIAIAVHSQT TDIPPCPHGWISLWKGFSFIMFTSAGSEGTGQALASPGSCLEEFRASPFLECHGRGTCNYYSNSYSFW LASLNPERMFRKPIPSTVKAGELEKIISRCQVCMKKRH Exemplary COL4A4 amino acid sequence – Uniprot reference P53420 (SEQ ID NO: 4) MWSLHIVLMRCSFRLTKSLATGPWSLILILFSVQYVYGSGKKYIGPCGGRDCSVCHCVPEKGSRGPPG PPGPQGPIGPLGAPGPIGLSGEKGMRGDRGPPGAAGDKGDKGPTGVPGFPGLDGIPGHPGPPGPRGKP GMSGHNGSRGDPGFPGGRGALGPGGPLGHPGEKGEKGNSVFILGAVKGIQGDRGDPGLPGLPGSWGAG GPAGPTGYPGEPGLVGPPGQPGRPGLKGNPGVGVKGQMGDPGEVGQQGSPGPTLLVEPPDFCLYKGEK GIKGIPGMVGLPGPPGRKGESGIGAKGEKGIPGFPGPRGDPGSYGSPGFPGLKGELGLVGDPGLFGLI GPKGDPGNRGHPGPPGVLVTPPLPLKGPPGDPGFPGRYGETGDVGPPGPPGLLGRPGEACAGMIGPPG PQGFPGLPGLPGEAGIPGRPDSAPGKPGKPGSPGLPGAPGLQGLPGSSVIYCSVGNPGPQGIKGKVGP PGGRGPKGEKGNEGLCACEPGPMGPPGPPGLPGRQGSKGDLGLPGWLGTKGDPGPPGAEGPPGLPGKH GASGPPGNKGAKGDMVVSRVKGHKGERGPDGPPGFPGQPGSHGRDGHAGEKGDPGPPGDHEDATPGGK GFPGPLGPPGKAGPVGPPGLGFPGPPGERGHPGVPGHPGVRGPDGLKGQKGDTISCNVTYPGRHGPPG FDGPPGPKGFPGPQGAPGLSGSDGHKGRPGTPGTAEIPGPPGFRGDMGDPGFGGEKGSSPVGPPGPPG SPGVNGQKGIPGDPAFGHLGPPGKRGLSGVPGIKGPRGDPGCPGAEGPAGIPGFLGLKGPKGREGHAG FPGVPGPPGHSCERGAPGIPGQPGLPGYPGSPGAPGGKGQPGDVGPPGPAGMKGLPGLPGRPGAHGPP GLPGIPGPFGDDGLPGPPGPKGPRGLPGFPGFPGERGKPGAEGCPGAKGEPGEKGMSGLPGDRGLRGA KGAIGPPGDEGEMAIISQKGTPGEPGPPGDDGFPGERGDKGTPGMQGRRGEPGRYGPPGFHRGEPGEK GQPGPPGPPGPPGSTGLRGFIGFPGLPGDQGEPGSPGPPGFSGIDGARGPKGNKGDPASHFGPPGPKG EPGSPGCPGHFGASGEQGLPGIQGPRGSPGRPGPPGSSGPPGCPGDHGMPGLRGQPGEMGDPGPRGLQ GDPGIPGPPGIKGPSGSPGLNGLHGLKGQKGTKGASGLHDVGPPGPVGIPGLKGERGDPGSPGISPPG PRGKKGPPGPPGSSGPPGPAGATGRAPKDIPDPGPPGDQGPPGPDGPRGAPGPPGLPGSVDLLRGEPG DCGLPGPPGPPGPPGPPGYKGFPGCDGKDGQKGPVGFPGPQGPHGFPGPPGEKGLPGPPGRKGPTGLP GPRGEPGPPADVDDCPRIPGLPGAPGMRGPEGAMGLPGMRGPSGPGCKGEPGLDGRRGVDGVPGSPGP PGRKGDTGEDGYPGGPGPPGPIGDPGPKGFGPGYLGGFLLVLHSQTDQEPTCPLGMPRLWTGYSLLYL EGQEKAHNQDLGLAGSCLPVFSTLPFAYCNIHQVCHYAQRNDRSYWLASAAPLPMMPLSEEAIRPYVS RCAVCEAPAQAVAVHSQDQSIPPCPQTWRSLWIGYSFLMHTGAGDQGGGQALMSPGSCLEDFRAAPFL ECQGRQGTCHFFANKYSFWLTTVKADLQFSSAPAPDTLKESQAQRQKISRCQVCVKYS Exemplary COL4A5 amino acid sequence – Uniprot reference P29400 (SEQ ID NO: 5) MKLRGVSLAAGLFLLALSLWGQPAEAAACYGCSPGSKCDCSGIKGEKGERGFPGLEGHPGLPGFPGPE GPPGPRGQKGDDGIPGPPGPKGIRGPPGLPGFPGTPGLPGMPGHDGAPGPQGIPGCNGTKGERGFPGS PGFPGLQGPPGPPGIPGMKGEPGSIIMSSLPGPKGNPGYPGPPGIQGLPGPTGIPGPIGPPGPPGLMG PPGPPGLPGPKGNMGLNFQGPKGEKGEQGLQGPPGPPGQISEQKRPIDVEFQKGDQGLPGDRGPPGPP GIRGPPGPPGGEKGEKGEQGEPGKRGKPGKDGENGQPGIPGLPGDPGYPGEPGRDGEKGQKGDTGPPG PPGLVIPRPGTGITIGEKGNIGLPGLPGEKGERGFPGIQGPPGLPGPPGAAVMGPPGPPGFPGERGQK GDEGPPGISIPGPPGLDGQPGAPGLPGPPGPAGPHIPPSDEICEPGPPGPPGSPGDKGLQGEQGVKGD KGDTCFNCIGTGISGPPGQPGLPGLPGPPGSLGFPGQKGEKGQAGATGPKGLPGIPGAPGAPGFPGSK GEPGDILTFPGMKGDKGELGSPGAPGLPGLPGTPGQDGLPGLPGPKGEPGGITFKGERGPPGNPGLPG LPGNIGPMGPPGFGPPGPVGEKGIQGVAGNPGQPGIPGPKGDPGQTITQPGKPGLPGNPGRDGDVGLP GDPGLPGQPGLPGIPGSKGEPGIPGIGLPGPPGPKGFPGIPGPPGAPGTPGRIGLEGPPGPPGFPGPK GEPGFALPGPPGPPGLPGFKGALGPKGDRGFPGPPGPPGRTGLDGLPGPKGDVGPNGQPGPMGPPGLP GIGVQGPPGPPGIPGPIGQPGLHGIPGEKGDPGPPGLDVPGPPGERGSPGIPGAPGPIGPPGSPGLPG KAGASGFPGTKGEMGMMGPPGPPGPLGIPGRSGVPGLKGDDGLQGQPGLPGPTGEKGSKGEPGLPGPP GPMDPNLLGSKGEKGEPGLPGIPGVSGPKGYQGLPGDPGQPGLSGQPGLPGPPGPKGNPGLPGQPGLI GPPGLKGTIGDMGFPGPQGVEGPPGPSGVPGQPGSPGLPGQKGDKGDPGISSIGLPGLPGPKGEPGLP GYPGNPGIKGSVGDPGLPGLPGTPGAKGQPGLPGFPGTPGPPGPKGISGPPGNPGLPGEPGPVGGGGH PGQPGPPGEKGKPGQDGIPGPAGQKGEPGQPGFGNPGPPGLPGLSGQKGDGGLPGIPGNPGLPGPKGE PGFHGFPGVQGPPGPPGSPGPALEGPKGNPGPQGPPGRPGLPGPEGPPGLPGNGGIKGEKGNPGQPGL PGLPGLKGDQGPPGLQGNPGRPGLNGMKGDPGLPGVPGFPGMKGPSGVPGSAGPEGEPGLIGPPGPPG LPGPSGQSIIIKGDAGPPGIPGQPGLKGLPGPQGPQGLPGPTGPPGDPGRNGLPGFDGAGGRKGDPGL PGQPGTRGLDGPPGPDGLQGPPGPPGTSSVAHGFLITRHSQTTDAPQCPQGTLQVYEGFSLLYVQGNK RAHGQDLGTAGSCLRRFSTMPFMFCNINNVCNFASRNDYSYWLSTPEPMPMSMQPLKGQSIQPFISRC AVCEAPAVVIAVHSQTIQIPHCPQGWDSLWIGYSFMMHTSAGAEGSGQALASPGSCLEEFRSAPFIEC HGRGTCNYYANSYSFWLATVDVSDMFSKPQSETLKAGDLRTRISRCQVCMKRT An example transgene encoding COL4A3 is provided in NM_000091.5. An example transgene encoding COL4A4 is provided in NM_000092.5. An example transgene encoding COL4A5 is provided in NM_000495.5. Suitably, the COL4A3 transgene may comprise or consist of the polynucleotide sequence shown as SEQ ID NO: 6, or a variant which is at least 70% identical to SEQ ID NO: 6. Suitably, the variant may be at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99% identical to SEQ ID NO: 6. Suitably, the COL4A4 transgene may comprise or consist of the polynucleotide sequence shown as SEQ ID NO: 7, or a variant which is at least 70% identical to SEQ ID NO: 7. Suitably, the variant may be at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99% identical to SEQ ID NO: 7. Suitably, the COL4A5 transgene may comprise or consist of the polynucleotide sequence shown as SEQ ID NO: 8, or a variant which is at least 70% identical to SEQ ID NO: 8. Suitably, the variant may be at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99% identical to SEQ ID NO: 8. Exemplary COL4A3 transgene sequence (SEQ ID NO: 6) atgagcgcccggaccgcccccaggccgcaggtgctcctgctgccgctcctgctggtgctcctggcggc ggcgcccgcagccagcaagggttgtgtctgtaaagacaaaggccagtgcttctgtgacggggccaaag gggagaagggggagaagggctttcctggaccccccggttctcctggccagaaaggattcacaggtcct gaaggcttgcctggaccgcagggacccaagggctttccaggacttccaggactcacgggttccaaagg tgtaaggggaataagtggattgccaggattttctggttctcctggacttccaggcaccccaggcaata ccgggccttacggacttgtcggtgtaccaggatgcagtggttctaagggtgagcaggggtttccagga ctcccagggacactgggctacccagggatcccgggtgctgctggtttgaaaggacaaaagggtgctcc tgctaaagaagaagatatagaacttgatgcaaaaggcgaccccgggttgccaggggctccaggacccc agggtttgccaggccctccaggttttcctgggcctgttggcccacctggtcctccgggattctttggc tttccaggagccatgggacctagaggacctaagggtcacatgggtgaaagagtgataggacataaagg agagcggggtgtgaaagggttaacaggacccccgggaccaccaggaacagttattgtgaccctaactg gcccagataacagaacggacctcaagggggaaaagggagacaagggagcaatgggcgagcctggacct cctggaccctcaggactgcctggagaatcatatggatctgaaaagggtgctcctggagaccctggcct gcagggaaaacccggaaaagatggtgttcctggcttccctggaagtgagggagtcaagggcaacaggg gtttccctgggttaatgggtgaagatggcattaagggacagaaaggggacattggccctccaggattt cgtggtccaacagaatattatgacacataccaggaaaagggagatgaaggcactccaggcccaccagg gcccagaggagctcgtggcccacaaggtcccagtggtccccccggagttcctggaagtcctggatcat caaggcctggcctcagaggagcccctggatggccaggcctgaaaggaagtaaaggggaacgaggccgc ccaggaaaggatgccatggggactcctgggtccccaggttgtgctggttcaccaggtcttccaggatc accgggacctccaggaccgccaggtgacatcgtttttcgcaagggtccacctggagatcacggactgc caggctatctagggtctccaggaatcccaggagttgatgggcccaaaggagaaccaggcctcctgtgt acacagtgcccttatatcccagggcctcccggtctcccaggattgccagggttacatggtgtaaaagg aatcccaggaagacaaggcgcagctggcttgaaaggaagcccagggtccccaggaaatacaggtcttc caggatttccaggtttcccaggtgcccagggtgacccaggacttaaaggagaaaaaggtgaaacactt cagcctgaggggcaagtgggtgtcccaggtgacccggggctcagaggccaacctgggagaaagggctt ggatggaattcctggaactccgggagtgaaaggattaccaggacctaaaggcgaactggctctgagtg gtgagaaaggggaccaaggtcctccaggggatcctggctcccctgggtccccaggacctgcaggacca gctggaccacctggctacggaccccaaggagaacctggtctccagggcacgcaaggagttcctggagc ccccggaccacccggagaagccggccctaggggagagctcagtgtttcaacaccagttccaggcccac caggacctccagggccccctggccatcctggcccccaaggtccacctggtatccctggatccctgggg aaatgtggagatcctggtcttccagggcctgatggtgaaccaggaattccaggaattggatttcctgg gcctcctggacctaagggagaccaaggttttccaggtacaaaaggatcactgggttgtcctggaaaaa tgggagagcctgggttacctggaaagccaggcctcccaggagccaagggagaaccagcagtagccatg cctggaggaccaggaacaccaggttttccaggagaaagaggcaattctggggaacatggagaaattgg actccctggacttccaggtctccctggaactccaggaaatgaagggcttgatggaccacgaggagatc cagggcagcctggaccacctggagaacaaggacccccaggaaggtgcatagagggtcccaggggagcc caaggacttccaggcttaaatggattgaaagggcaacaaggcagaagaggtaaaacggggccaaaggg agacccaggaattccaggcttggatagatcaggatttcctggagaaactggatcaccaggaattccag gtcatcaaggtgaaatgggaccactgggtcaaagaggatatccaggaaatccgggaattttagggcca ccaggtgaagatggagtgattgggatgatgggctttcctggagccattggccctccagggccccctgg gaacccaggcacaccagggcagagggggagccctggaattccaggagtaaagggccagagaggaaccc caggagccaagggggaacaaggagataaaggaaatcccgggccttcagagatatcccacgtaataggg gacaaaggagaaccaggtctcaaaggattcgcaggaaatccaggtgagaaaggaaacagaggcgttcc agggatgccaggtttaaagggcctcaaaggactacccggaccagcaggaccaccaggccccagaggag atttgggcagcactgggaatcctggagaaccaggactgcgtggtataccaggaagcatggggaacatg ggcatgccaggttctaaaggaaaaaggggaactttgggattcccaggtcgagcaggaagaccaggcct cccaggtattcatggtctccagggagataagggagagccaggttattcagaaggtacaaggccaggac caccgggaccaacgggggatccaggactgccgggtgatatgggaaagaaaggagaaatggggcaacct ggcccacctggacatttggggcctgctggacctgagggagcccctggaagtcctggaagtcctggcct cccaggaaagccaggtcctcatggtgatttgggttttaaaggaatcaaaggcctcctgggccctccag gaatcagaggccctccaggtcttccaggatttccaggatctcctggaccaatgggtataagaggtgac caaggacgtgatggaattcctggtccagccggagaaaagggagaaacgggtttattgagggcccctcc aggcccaagagggaaccctggtgctcaaggagccaaaggagacaggggagccccaggttttcctggcc tcccgggcagaaaaggggccatgggagatgctggacctcgaggacccacaggcatagaaggattccca gggccaccaggtctgcccggtgcaattatccctggccagacaggaaatcgtggtccaccaggctcaag aggaagcccaggtgcgcctggtccccctggacctccagggagtcatgtaataggcataaaaggagaca aagggtctatgggccaccctggcccaaaaggtccacctggaactgcaggagacatgggaccaccaggt cgtctgggagcaccaggtactccaggtcttccaggacccagaggtgatcctggattccaggggtttcc aggcgtgaaaggagaaaagggtaatcctggatttctaggatccattggacctccaggaccaattgggc caaaaggaccacctggtgtacgtggagaccctggcacacttaagattatctcccttccaggaagccca gggccacctggcacacctggagaaccagggatgcagggagaacctgggccaccagggccacctggaaa cctaggaccctgtgggccaagaggtaagccaggcaaggatggaaaaccaggaactcctggaccagctg gagaaaaaggcaacaaaggttctaaaggagagccaggaccagctggatcagatggattgccaggtttg aaaggaaaacgtggagacagtggatcacctgcaacctggacaacgagaggctttgtcttcacccgaca cagtcaaaccacagcaattccttcatgtccagaggggacagtgccactctacagtgggttttcttttc tttttgtacaaggaaatcaacgagcccacggacaagaccttggaactcttggcagctgcctgcagcga tttaccacaatgccattcttattctgcaatgtcaatgatgtatgtaattttgcatctcgaaatgatta ttcatactggctgtcaacaccagctctgatgccaatgaacatggctcccattactggcagagcccttg agccttatataagcagatgcactgtttgtgaaggtcctgcgatcgccatagccgttcacagccaaacc actgacattcctccatgtcctcacggctggatttctctctggaaaggattttcattcatcatgttcac aagtgcaggttctgagggcaccgggcaagcactggcctcccctggctcctgcctggaagaattccgag ccagcccatttctagaatgtcatggaagaggaacgtgcaactactattcaaattcctacagtttctgg ctggcttcattaaacccagaaagaatgttcagaaagcctattccatcaactgtgaaagctggggaatt agaaaaaataataagtcgctgtcaggtgtgcatgaagaaaagacactga Exemplary COL4A4 transgene sequence (SEQ ID NO: 7) atgtggtctctgcacatagtactaatgaggtgctccttcagattgaccaagtccttggccacaggtcc ctggtcacttatactcattctcttttctgtacaatatgtatatgggagtggaaagaaatacattggtc cttgtggaggaagagattgctctgtttgccactgtgttcctgaaaaggggtctcggggtccaccagga ccaccagggccacagggtccaattggacccctgggagccccaggacccattgggctttcaggagagaa aggaatgagaggggaccgcggccctcctggagcagcaggggacaaaggagataagggtccaactggtg ttcctggatttccaggtttagatggcatacctgggcacccagggcctcctggacccagaggcaaacct ggtatgagtggccacaatggctcaagaggtgacccagggtttccaggaggaagaggagctcttggccc aggaggccccctaggccatcctggggaaaagggagaaaaaggaaattcagtgttcattttaggtgccg ttaaaggtattcagggagacagaggggacccaggactgcctggcttaccaggatcttggggtgcagga ggaccggcaggtcccacaggatatcctggagagccagggttagtgggacctccgggccaaccagggcg tccaggtttgaagggaaatcccggtgtgggagtaaaggggcaaatgggagacccgggtgaggttggtc agcaaggttctcctggacccaccctgttggtagagccacctgacttttgtctctataaaggagaaaag ggtataaaaggaattcctggaatggttggactgccaggaccaccaggacgcaagggagaatctggtat tggggcaaaaggagaaaaaggtattcctggatttccagggcctcggggggatcctggttcctatggat ctccaggttttccaggattaaagggagaactaggactggttggagatcctgggctatttggattaatt ggcccaaagggggatcctggaaatcgagggcacccaggaccaccaggtgttttggtgactccacctct tccactcaaaggcccaccaggggacccagggttccctggccgctatggagaaacaggggatgttggac cacctggtcccccaggtctcttgggcagaccaggggaagcctgtgcaggcatgataggaccccctggg ccacaaggatttcctggtcttcctgggcttccaggagaagctggtattcctgggagacctgattctgc tccaggaaaaccagggaagccaggatcacctggcttgcctggagcaccaggcctgcagggcctcccag gatcaagtgtgatatactgtagtgttgggaaccccggaccacaaggaataaaaggcaaagttggtccc ccaggaggaagaggcccaaaaggagaaaaaggaaatgaaggactctgtgcctgtgagcctggacccat gggcccccctggccctccaggacttcctgggaggcaggggagtaagggagacttggggctccctggct ggcttggaacaaaaggtgacccaggacctcctggtgctgaaggacctccagggctaccaggaaagcat ggtgcctctggaccacctggcaacaaaggggcgaagggtgacatggttgtatcaagagttaaagggca caaaggagaaagaggtcctgatgggcccccaggatttccagggcagccaggatcacatggtcgggatg gacatgctggagaaaaaggggatccaggacctccaggggatcatgaagatgcgaccccaggtggtaaa ggatttcctggacctctgggccccccaggcaaagcaggacctgtggggcccccaggactgggatttcc tggtccaccaggagagcgaggccacccaggagttccaggccacccaggtgtgaggggccctgatggct tgaagggtcagaaaggtgacacaatttcttgcaacgtaacctaccctgggaggcatggccctccaggt tttgatggacctccaggtccgaagggatttccaggtccccaaggtgcccctgggctgagtggttcaga tgggcataaaggcagacctggcacaccaggaacagcggaaataccaggtccacctggttttcgtggtg acatgggagatccgggttttggaggtgaaaaggggtcctcccctgttgggcccccaggccctcccggc tcaccaggagtgaatggtcagaaaggaatcccgggagaccctgcatttggtcacctgggacccccggg aaagaggggtctttcaggagtgccagggataaaaggacccagaggtgatccgggatgtccaggggctg aagggccagctggcattcctggattcctaggtctcaaaggtcccaaaggcagagagggacatgctggg tttccaggtgtcccaggtccacctggccattcctgtgaaagaggtgctccagggataccagggcaacc gggactccctgggtatccaggtagcccaggtgctccaggtgggaaaggacagccgggagatgtggggc ctcccgggccagctggaatgaaaggcctccccggactcccaggacggcctggggcacatggtccccca ggcctcccaggaatcccaggtccctttggagatgatgggctacctggtcctccaggtccaaagggacc ccgggggctgcctggtttcccaggttttcccggagaaagaggaaagcctggtgcagagggatgtcctg gcgcaaagggagaacctggagagaagggcatgtctggccttcctggagaccggggactgagaggggcc aaaggagccataggacctcccggagatgaaggagaaatggctatcatttcacaaaagggaacacctgg ggaacctggacctcctggagatgatggattcccaggagaaagaggtgataaaggaactcccgggatgc aagggagaagaggagagccgggaagatacggaccacctggatttcacagaggggaacctggtgagaaa ggtcagccagggcctcctggacccccaggccctccaggctcaactggtctaagagggttcattggttt tccaggacttccaggtgaccagggtgagccaggttctccaggtccccctggattttcaggaattgatg gagcaagaggacctaaaggaaacaaaggtgaccctgccagtcactttggtccacctggtccaaagggt gagccaggtagccctggatgtccagggcattttggagcatccggagagcagggcttgcctggtattca agggcccagaggatcacctggaaggccagggccacctggctcctctggaccaccagggtgcccaggtg atcacgggatgcctgggctgaggggacagccaggagaaatgggagaccctgggccaagaggcctccag ggggatccagggataccaggtcctccgggaataaaaggtccctccggatcacctggcctgaacggctt gcatggattgaaaggtcagaaaggaactaaaggtgcttcaggtttgcatgatgtggggccacctggtc cagtgggaatacctgggctaaaaggggagagaggagaccctgggagcccaggaatctctcctccaggt cctcgtggaaagaaaggtcccccaggacccccagggagttcaggaccacctggtcctgcaggtgccac aggaagagctcctaaggacattcctgacccgggtccacctggagatcagggacctcctggtcctgatg gcccaagaggagcacctgggcctccaggcctccctgggagtgttgaccttctgagaggggagccaggt gactgtggtctaccagggccaccaggtccccctggcccaccaggccctccaggatacaaaggctttcc aggatgtgatggaaaagatggccagaaaggaccagtgggattcccgggaccgcagggaccacatggat ttcctgggccacctggagagaagggtttacctggacctccagggagaaaagggcccactggtcttccg ggtcccagaggtgaaccggggccacctgcagatgtggatgactgtccccgaatcccaggccttcctgg ggcgccaggcatgagaggaccagaaggagccatggggctccctggaatgagaggcccctcaggaccag ggtgcaaaggagagcctgggctggatggcaggaggggtgtggatggcgtccctgggtctcctgggcct cccggacgtaaaggtgacacaggagaagacggctaccctggaggaccagggcctcctggtcccattgg ggatcctgggcccaaagggtttggccctggatacctcggtggcttcctcctggttctccacagtcaga cggaccaggagcccacctgccccctgggcatgcccaggctctggactgggtatagtctgttatacctg gaagggcaagagaaagctcacaatcaagaccttggtctggcagggtcttgccttcccgtatttagcac gctgccctttgcctactgcaacatccaccaggtgtgccactatgcccagagaaacgacagatcctact ggctggccagcgctgcgcccctccccatgatgccactctctgaagaggcgatccgcccctatgtcagc cgctgtgcggtatgcgaggccccggcccaggcggtggcggtgcacagccaggaccagtccatcccccc atgtccgcagacctggaggagcctctggatcgggtattcattcctgatgcacacaggagctggggacc aaggaggagggcaggcccttatgtcacctggcagctgcctggaagatttcagagcagcaccattcctt gaatgccagggccggcagggaacttgccactttttcgcaaataagtatagcttctggctcacaacggt gaaagcagacttgcagttttcctctgctccagcaccagacaccttaaaagaaagccaggcccaacgcc agaaaatcagccggtgccaggtctgcgtgaagtatagctag Exemplary COL4A5 transgene sequence (SEQ ID NO: 8) atgaaactgcgtggagtcagcctggctgccggcttgttcttactggccctgagtctttgggggcagcc tgcagaggctgcggcttgctatgggtgttctccaggatcaaagtgtgactgcagtggcataaaagggg aaaagggagagagagggtttccaggtttggaaggacacccaggattgcctggatttccaggtccagaa gggcctccggggcctcggggacaaaagggtgatgatggaattccagggccaccaggaccaaaaggaat cagaggtcctcctggacttcctggatttccagggacaccaggtcttcctggaatgccaggccacgatg gggccccaggacctcaaggtattcccggatgcaatggaaccaagggagaacgtggatttccaggcagt cccggttttcctggtttacagggtcctccaggaccccctgggatcccaggtatgaagggtgaaccagg tagtataattatgtcatcactgccaggaccaaagggtaatccaggatatccaggtcctcctggaatac aaggcctacctggtcccactggtataccagggccaattggtcccccaggaccaccaggtttgatgggc cctcctggtccaccaggacttccaggacctaaggggaatatgggcttaaatttccagggacccaaagg tgaaaaaggtgagcaaggtcttcagggcccacctgggccacctgggcagatcagtgaacagaaaagac caattgatgtagagtttcagaaaggagatcagggacttcctggtgaccgagggcctcctggacctcca gggatacgtggtcctccaggtcccccaggtggtgagaaaggtgagaagggtgagcaaggagagccagg caaaagaggtaaaccaggcaaagatggagaaaatggccaaccaggaattcctggtttgcctggtgatc ctggttaccctggtgaacccggaagggatggtgaaaagggccaaaaaggtgacactggcccacctgga cctcctggacttgtaattcctagacctgggactggtataactataggagaaaaaggaaacattgggtt gcctgggttgcctggagaaaaaggagagcgaggatttcctggaatacagggtccacctggccttcctg gacctccaggggctgcagttatgggtcctcctggccctcctggatttcctggagaaaggggtcagaaa ggtgatgaaggaccacctggaatttccattcctggacctcctggacttgacggacagcctggggctcc tgggcttccagggcctcctggccctgctggccctcacattcctcctagtgatgagatatgtgaaccag gccctccaggccccccaggatctccaggtgataaaggactccaaggagaacaaggagtgaaaggtgac aaaggtgacacttgcttcaactgcattggaactggtatttcagggcctccaggtcaacctggtttgcc aggtctcccaggtcctccaggatctcttggtttccctggacagaaaggggaaaaaggacaagctggtg caactggtcccaaaggattaccaggcattccaggagctccaggtgctccaggctttcctggatctaaa ggtgaacctggtgatatcctcacttttccaggaatgaagggtgacaaaggagagttgggttcccctgg agctccagggcttcctggtttacctggcactcctggacaggatggattgccagggcttcctggcccga aaggagagcctggtggaattacttttaagggtgaaagaggtccccctgggaacccaggtttaccaggc ctcccagggaatatagggcctatgggtccccctggtttcggccctccaggcccagtaggtgaaaaagg catacaaggtgtggcaggaaatccaggccagccaggaataccaggtcctaaaggggatccaggtcaga ctataacccagccggggaagcctggcttgcctggtaacccaggcagagatggtgatgtaggtcttcca ggtgaccctggacttccagggcaaccaggcttgccagggatacctggtagcaaaggagaaccaggtat ccctggaattgggcttcctggaccacctggtcccaaaggctttcctggaattccaggacctccaggag cacctgggacacctggaagaattggtctagaaggccctcctgggccacccggctttccaggaccaaag ggtgaaccaggatttgcattacctgggccacctgggccaccaggacttccaggtttcaaaggagcact tggtccaaaaggtgatcgtggtttcccaggacctccgggtcctccaggacgcactggcttagatgggc tccctggaccaaaaggtgatgttggaccaaatggacaacctggaccaatgggacctcctgggctgcca ggaataggtgttcagggaccaccaggaccaccagggattcctgggccaataggtcaacctggtttaca tggaataccaggagagaagggggatccaggacctcctggacttgatgttccaggacccccaggtgaaa gaggcagtccagggatccccggagcacctggtcctataggacctccaggatcaccagggcttccagga aaagcaggtgcctctggatttccaggtaccaaaggtgaaatgggtatgatgggacctccaggcccacc aggacctttgggaattcctggcaggagtggtgtacctggtcttaaaggtgatgatggcttgcagggtc agccaggacttcctggccctacaggagaaaaaggtagtaaaggagagcctggccttccaggccctcct ggaccaatggatccaaatcttctgggctcaaaaggagagaagggggaacctggcttaccaggtatacc tggagtttcagggccaaaaggttatcagggtttgcctggagacccagggcaacctggactgagtggac aacctggattaccaggaccaccaggtcccaaaggtaaccctggtctccctggacagccaggtcttata ggacctcctggacttaaaggaaccatcggtgatatgggttttccagggcctcagggtgtggaagggcc tcctggaccttctggagttcctggacaacctggctccccaggattacctggacagaaaggcgacaaag gtgatcctggtatttcaagcattggtcttccaggtcttcctggtccaaagggtgagcctggtctgcct ggatacccagggaaccctggtatcaaaggttctgtgggagatcctggtttgcccggattaccaggaac ccctggagcaaaaggacaaccaggccttcctggattcccaggaaccccaggccctcctggaccaaaag gtattagtggccctcctgggaaccccggccttccaggagaacctggtcctgtaggtggtggaggtcat cctgggcaaccagggcctccaggcgaaaaaggcaaacccggtcaagatggtattcctggaccagctgg acagaagggtgaaccaggtcaaccaggctttggaaacccaggaccccctggacttccaggactttctg gccaaaagggtgatggaggattacctgggattccaggaaatcctggccttccaggtccaaagggcgaa ccaggctttcacggtttccctggtgtgcagggtcccccaggccctcctggttctccgggtccagctct ggaaggacctaaaggcaaccctgggccccaaggtcctcctgggagaccaggtctaccaggtccagaag gtcctccaggtctccctggaaatggaggtattaaaggagagaagggaaatccaggccaacctgggcta cctggcttgcctggtttgaaaggagatcaaggaccaccaggactccagggtaatcctggccggccggg tctcaatggaatgaaaggagatcctggtctccctggtgttccaggattcccaggcatgaaaggaccca gtggagtacctggatcagctggccctgagggggaaccgggacttattggtcctccaggtcctcctgga ttacctggtccttcaggacagagtatcataattaaaggagatgctggtcctccaggaatccctggcca gcctgggctaaagggtctaccaggaccccaaggacctcaaggcttaccaggtccaactggccctccag gagatcctggacgcaatggactccctggctttgatggtgcaggagggcgcaaaggagacccaggtctg ccaggacagccaggtacccgtggtttggatggtccccctggtccagatggattgcaaggtcccccagg tccccctggaacctcctctgttgcacatggatttcttattacacgccacagccagacaacggatgcac cacaatgcccacagggaacacttcaggtctatgaaggcttttctctcctgtatgtacaaggaaataaa agagcccacggtcaagacttggggacggctggcagctgccttcgtcgctttagtaccatgcctttcat gttctgcaacatcaataatgtttgcaactttgcttcaagaaatgactattcttactggctctctaccc cagagcccatgccaatgagcatgcaacccctaaagggccagagcatccagccattcattagtcgatgt gcagtatgtgaagctccagctgtggtgatcgcagttcacagtcagacgatccagattccccattgtcc tcagggatgggattctctgtggattggttattccttcatgatgcatacaagtgcaggggcagaaggct caggtcaagccctagcctcccctggttcctgcttggaagagtttcgttcagctcccttcatcgaatgt catgggaggggtacctgtaactactatgccaactcctacagcttttggctggcaactgtagatgtgtc agacatgttcagtaaacctcagtcagaaacgctgaaagcaggagacttgaggacacgaattagccgat gtcaagtgtgcatgaagaggacataa The COL4A3, COL4A4 or COL4A5 transgene may comprise an intron or intronic sequences, which can be used to improve gene expression. The COL4A3, COL4A4 or COL4A5 transgene may comprise a protein tag, such as a hemagglutinin (HA) tag. HA can be used as an epitope tag and has been shown not to interfere with bioactivity or biodistribution of proteins to which it has been added. The protein tag can facilitate detection, isolation, and purification of the transgene. Other suitable protein tags may include Myc tags, polyhistidine tags and flag tags. Nephrotic syndrome (NS)-associated transgene The transgene may comprise or consist of a NS-associated gene. Nephrotic syndrome (NS) is a chronic kidney disease characterized by significant proteinuria, hypoalbuminemia, oedema and hyperlipidemia. The NS-associated transgene may be a gene associated with a monogenic form of NS and expressed in podocytes. Suitable NS-associated transgenes include NPHS2, ADCK4, ALG1, ARHGAP24, ARGHDIA, CD151, CD2AP, COQ2, COQ6, DGKE, E2F3, EMP2, KANK2, LAGE3, LMNA, LMX1B, MAFB, NUP85, NUP93, NXF5, OSGEP, PAX2, PDSS2, PMM2, PODXL, SCARB2, SGPL1, Smad7, TP53RK, TPRKB, VDR, WDR73, WT1, ZMPSTE24, APOL1, NPHS1, TRPC6, NUP107, NUP133, NUP160, ACTN4, INF2, ANKFY1, ANLN, CRB2, ITGA3, KANK1, KANK4, MAGI2, MYO1E, OCRL, PTPRO, SMARCAL1, SYNPO, TBC1D8B, XPO5, TNS2 and NLRP3. NPHS2 The transgene may encode NPHS2, or a fragment and/or variant thereof. “NPHS2” is the abbreviated name of the polypeptide encoded by the NPHS2 gene and is also known as podocin. NPHS2 is a 42kDa hairpin like membrane-associated podocyte-specific protein that is a key component of the protein complex at the slit diaphragm; the cell-cell junction between adjacent podocyte foot processes. It localises to lipid rafts and interacts with other important slit diaphragm proteins like nephrin, CD2AP and TRPC6. It is essential in the maintenance of the slit diaphragm, and consequently the integrity of the glomerular filtration barrier. A fragment and/or variant of NPHS2 may retain NPHS2 function. For example, a fragment and/or variant of podocin may regulate glomerular permeability. Suitably, a fragment and/or variant of NPHS2 may have the same or similar function to NPHS2, e.g. may have at least 40%, at least 50%, at least 60%, at least 70%, at least 80%, at least 90%, at least 95%, or at least 100% of the function of NPHS2. A person skilled in the art would be able to generate fragments and/or variants using conservative substitutions, based on the known structural and functional features of NPHS2 (see e.g. Tabassum, A., et al., 2014. Interdisciplinary Sciences: Computational Life Sciences, 6(1), pp.32-39), and/or based on known variants (see e.g. NCBI Gene ID: 7827 and NCBI HomoloGene: 22826). Suitably, a fragment and/or variant of NPHS2 comprises a transmembrane domain, with two cytoplasmic domains at the N- and C-terminus. The NPHS2 gene is conserved in chimpanzee, Rhesus monkey, dog, cow, mouse, and rat. The NPHS2 may be a human NPHS2. Suitably, the NPHS2 may comprise or consist of a polypeptide sequence of UniProtKB accession Q9NP85, or a fragment and/or variant thereof. In some embodiments, the NPHS2 polypeptide comprises or consists of an amino acid sequence which is at least 70% identical to SEQ ID NO: 9 or a fragment thereof. Suitably, the NPHS2 polypeptide comprises or consists of an amino acid sequence which is at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 9 or a fragment thereof. In some embodiments, the NPHS2 polypeptide comprises or consists of SEQ ID NO: 9 or a fragment thereof. MERRARSSSRESRGRGGRTPHKENKRAKAERSGGGRGRQEAGPEPSGSGRAGTPGEPRAPAATVVDVDE VRGSGEEGTEVVALLESERPEEGTKSSGLGACEWLLVLISLLFIIMTFPFSIWFCVKVVQEYERVIIFR LGHLLPGRAKGPGLFFFLPCLDTYHKVDLRLQTLEIPFHEIVTKDMFIMEIDAICYYRMENASLLLSSL AHVSKAVQFLVQTTMKRLLAHRSLTEILLERKSIAQDAKVALDSVTCIWGIKVERIEIKDVRLPAGLQH SLAVEAEAQRQAKVRMIAAEAEKAASESLRMAAEILSGTPAAVQLRYLHTLQSLSTEKPSTVVLPLPFD LLNCLSSPSNRTQGSLPFPSPSKPVEPLNPKKKDSPML Exemplary NPHS2amino acid sequence (SEQ ID NO: 9) In some embodiments, the NPHS2 transgene comprises or consists of a nucleotide sequence which is at least 70% identical to SEQ ID NO: 10 or a fragment thereof. Suitably, the NPHS2 transgene comprises or consists of a nucleotide sequence which is at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 10 or a fragment thereof. In some embodiments, the NPHS2 transgene, comprises or consists of the nucleotide sequence SEQ ID NO: 10 or a fragment thereof. ATGGAGAGGAGGGCGCGGAGCTCCTCCAGGGAGTCCCGCGGGCGAGGCGGCAGGACTCCGCACAAGGAG AACAAGAGGGCAAAGGCCGAGAGGAGCGGCGGGGGCCGCGGGCGCCAGGAGGCTGGGCCCGAGCCGTCG GGCTCCGGACGGGCGGGGACCCCGGGGGAGCCCCGAGCGCCCGCCGCCACGGTGGTGGACGTGGATGAG GTCCGAGGCTCCGGCGAGGAGGGCACCGAGGTGGTGGCGCTGTTGGAGAGCGAGCGGCCCGAGGAAGGT ACCAAATCCTCCGGCTTAGGGGCCTGTGAGTGGCTTCTTGTCCTCATTTCCCTGCTCTTCATCATCATG ACCTTCCCTTTTTCCATCTGGTTCTGCGTAAAGGTTGTACAAGAGTATGAAAGAGTAATTATATTCCGA CTGGGACATCTGCTTCCTGGAAGAGCCAAAGGCCCTGGTCTTTTCTTTTTTTTGCCCTGCCTGGATACC TACCACAAGGTTGACCTTCGTCTCCAAACTCTGGAGATACCTTTTCATGAGATCGTGACCAAAGACATG TTTATAATGGAGATAGATGCCATTTGCTACTACCGAATGGAAAATGCCTCTCTTCTCCTAAGCAGTCTT GCTCATGTATCTAAAGCTGTGCAATTCCTTGTGCAAACCACTATGAAGCGTCTCCTAGCACATCGATCC CTCACTGAAATTCTTCTAGAGAGGAAGAGCATCGCCCAAGATGCAAAGGTTGCCTTGGATTCAGTGACC TGTATTTGGGGAATCAAAGTGGAGAGAATAGAAATTAAAGATGTGAGGTTGCCAGCTGGGCTTCAGCAC TCACTGGCTGTGGAGGCTGAAGCGCAAAGACAAGCCAAAGTGCGGATGATTGCTGCAGAAGCGGAAAAG GCTGCTTCTGAGTCCCTGAGGATGGCAGCTGAGATTCTGTCAGGCACCCCTGCTGCTGTTCAGCTTCGA TACCTCCACACCCTTCAGTCTCTGTCCACAGAGAAGCCTTCCACTGTGGTTTTACCTTTGCCATTTGAC CTACTGAATTGCCTGTCTTCTCCCAGCAACAGAACTCAGGGAAGCCTCCCCTTCCCAAGTCCTTCCAAA CCTGTTGAGCCACTAAATCCTAAAAAGAAAGACTCTCCCATGTTA Exemplary NPHS2 transgene sequence (SEQ ID NO: 10) In some embodiments, the NPHS2 transgene comprises or consists of a nucleotide sequence which is at least 70% identical to SEQ ID NO: 11 or a fragment thereof. Suitably, the NPHS2 transgene comprises or consists of a nucleotide sequence which is at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 11 or a fragment thereof. In some embodiments, the NPHS2 transgene, comprises or consists of the nucleotide sequence SEQ ID NO: 11 or a fragment thereof. ATGGAGAGGAGGGCGCGGAGCTCCTCCAGGGAGTCCCGCGGGCGAGGCGGCAGGACTCCGCACAAGGAG AACAAGAGGGCAAAGGCCGAGAGGAGCGGCGGAGGCCGCGGGCGCCAGGAGGCTGGGCCCGAGCCGTCG GGCTCCGGACGGGCGGGGACCCCGGGGGAGCCCCGAGCGCCCGCCGCCACGGTGGTGGACGTGGATGAG GTCCGAGGCTCCGGCGAGGAGGGCACCGAGGTGGTGGCGCTGTTGGAGAGCGAGCGGCCCGAGGAAGGT ACCAAATCCTCCGGCTTAGGGGCCTGTGAGTGGCTTCTTGTCCTCATTTCCCTGCTCTTCATCATCATG ACCTTCCCTTTTTCCATCTGGTTCTGCGTAAAGGTTGTACAAGAGTATGAAAGAGTAATTATATTCCGA CTGGGACATCTGCTTCCTGGAAGAGCCAAAGGCCCTGGTCTTTTCTTTTTTTTGCCCTGCCTGGATACC TACCACAAGGTTGACCTTCGTCTCCAAACTCTGGAGATACCTTTTCATGAGATCGTGACCAAAGACATG TTTATAATGGAGATAGATGCCATTTGCTACTACCGAATGGAAAATGCCTCTCTTCTCCTAAGCAGTCTT GCTCATGTATCTAAAGCTGTGCAATTCCTTGTGCAAACCACTATGAAGCGTCTCCTAGCACATCGATCC CTCACTGAAATTCTTCTAGAGAGGAAGAGCATCGCCCAAGATGCAAAGGTTGCCTTGGATTCAGTGACC TGTATTTGGGGAATCAAAGTGGAGAGAATAGAAATTAAAGATGTGAGGTTGCCAGCTGGGCTTCAGCAC TCACTGGCTGTGGAGGCTGAAGCGCAAAGACAAGCCAAAGTGCGGATGATTGCTGCAGAAGCGGAAAAG GCTGCTTCTGAGTCCCTGAGGATGGCAGCTGAGATTCTGTCAGGCACCCCTGCTGCCGTTCAGCTTCGA TACCTCCACACCCTTCAGTCTCTGTCCACAGAGAAGCCTTCCACTGTGGTTTTACCTTTGCCATTTGAC CTACTGAATTGCCTGTCTTCTCCCAGCAACAGAACTCAGGGAAGCCTCCCCTTCCCAAGTCCTTCCAAA CCTGTTGAGCCACTAAATCCTAAAAAGAAAGACTCTCCCATGTTATAG Exemplary NPHS2 transgene sequence (SEQ ID NO: 11) Vascular endothelial growth factor (VEGF)C transgene The transgene may comprise or consist of a vascular endothelial growth factor (VEGF)C transgene. VEGFC is a lymphangiogenic growth factor, which is known to signal via two receptors, VEGFR-3 (Flt4) and VEGFR-2 (Flk4). VEGFC is produced by cells in a prepropeptide form, which dimerises before being cleaved into a tetramer. The VEGFC transgene may comprise a polynucleotide encoding any form of VEGFC, such as the prepropeptide form, the tetramer form, the intermediate form, or the fully processed mature VEGFC. If desired, polynucleotides encoding different forms of VEGFC polypeptides may be used in any combination. Preferably, the VEGFC transgene comprises a polynucleotide encoding one or more polypeptides having VEGFC biological activity, i.e., peptides that can bind to and activate VEGFR-2 and/or VEGRF-3. More preferably, the VEGFC transgene comprises a polynucleotide encoding a polypeptide comprising the VEGFC homology domain and having VEGFC biological activity, i.e., a polypeptide that can bind to and activate VEGFR-2 and/or VEGRF-3. Further details of suitable VEGFC polynucleotides and polypeptides include those described in WO 2015/022447 and US 2014/0087002. The VEGFC polynucleotide may comprise the VEGFC open reading frame (ORF) sequence of SEQ ID NO: 12. The VEGFC polynucleotide may comprise a nucleic acid sequence which has at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, or at least 99% sequence identity to the VEGFC ORF sequence of SEQ ID NO: 12. The variant sequence may encode a VEGFC polypeptide that has retained the capability to bind and activate VEGFR-2 and VEGFR-3. Exemplary VEGFC polynucleotide (SEQ ID NO: 12) ATGCACTTGCTGGGCTTCTTCTCTGTGGCGTGTTCTCTGCTCGCCGCTGCGCTGCTCCCGGGTCCTCG CGAGGCGCCCGCCGCCGCCGCCGCCTTCGAGTCCGGACTCGACCTCTCGGACGCGGAGCCCGACGCGG GCGAGGCCACGGCTTATGCAAGCAAAGATCTGGAGGAGCAGTTACGGTCTGTGTCCAGTGTAGATGAA CTCATGACTGTACTCTACCCAGAATATTGGAAAATGTACAAGTGTCAGCTAAGGAAAGGAGGCTGGCA ACATAACAGAGAACAGGCCAACCTCAACTCAAGGACAGAAGAGACTATAAAATTTGCTGCAGCACATT ATAATACAGAGATCTTGAAAAGTATTGATAATGAGTGGAGAAAGACTCAATGCATGCCACGGGAGGTG TGTATAGATGTGGGGAAGGAGTTTGGAGTCGCGACAAACACCTTCTTTAAACCTCCATGTGTGTCCGT CTACAGATGTGGGGGTTGCTGCAATAGTGAGGGGCTGCAGTGCATGAACACCAGCACGAGCTACCTCA GCAAGACGTTATTTGAAATTACAGTGCCTCTCTCTCAAGGCCCCAAACCAGTAACAATCAGTTTTGCC AATCACACTTCCTGCCGATGCATGTCTAAACTGGATGTTTACAGACAAGTTCATTCCATTATTAGACG TTCCCTGCCAGCAACACTACCACAGTGTCAGGCAGCGAACAAGACCTGCCCCACCAATTACATGTGGA ATAATCACATCTGCAGATGCCTGGCTCAGGAAGATTTTATGTTTTCCTCGGATGCTGGAGATGACTCA ACAGATGGATTCCATGACATCTGTGGACCAAACAAGGAGCTGGATGAAGAGACCTGTCAGTGTGTCTG CAGAGCGGGGCTTCGGCCTGCCAGCTGTGGACCCCACAAAGAACTAGACAGAAACTCATGCCAGTGTG TCTGTAAAAACAAACTCTTCCCCAGCCAATGTGGGGCCAACCGAGAATTTGATGAAAACACATGCCAG TGTGTATGTAAAAGAACCTGCCCCAGAAATCAACCCCTAAATCCTGGAAAATGTGCCTGTGAATGTAC AGAAAGTCCACAGAAATGCTTGTTAAAAGGAAAGAAGTTCCACCACCAAACATGCAGCTGTTACAGAC GGCCATGTACGAACCGCCAGAAGGCTTGTGAGCCAGGATTTTCATATAGTGAAGAAGTGTGTCGTTGT GTCCCTTCATATTGGAAAAGACCACAAATGAGC Complement proteins and complement inhibitors The transgene may encode a complement protein, or a fragment and/or variant thereof. As used herein, a “complement protein” is a protein which is part of the complement system. The complement system, also known as complement cascade, is a central part of the innate immunity that serves as a first line of defence against foreign and altered host cells. The complement system is composed of plasma proteins produced mainly by the liver or membrane proteins expressed on cell surface. Complement operates in plasma, in tissues, or within cells. Complement proteins collaborate as a cascade to opsonize pathogens and induce a series of inflammatory responses helping immune cells to fight infection and maintain homeostasis (Merle, N.S., et al., 2015. Frontiers in immunology, 6, 262). There are three pathways of complement activation: the classical, the alternative, and the lectin pathways. The three complement pathways differ in their mechanisms of target recognition but converge in the activation of the central component C3. After this activation, C5 is cleaved, and the assembly of the membrane attack complex (MAC) is initiated. The enzymatic cleavage of C3 and C5 leads to the production and release of anaphylotoxins C3a and C5a. Suitably, the complement protein is selected from the list consisting of CFI, CFH, FHL-1, C1INH, C4BP, MASP2, C3, C5aR1, C5, C5a, CD55, CD35, CD46, CD59, vitronectin, and clusterin, or fragments and/or variants thereof. The transgene may encode an inhibitor of the complement system, or a fragment and/or variant thereof. As used herein, an “inhibitor of the complement system” or “complement inhibitor” is a protein which prevents activation of the complement system. Complement is tightly controlled by these inhibitors, which naturally protect self-cells and tissues from unwanted complement activation. Complement inhibitors can regulate complement activation in different stages of the classical, lectin, and alternative pathways. Suitably, the complement inhibitor is a naturally- occurring complement inhibitor, or a fragment and/or variant thereof. Preferably, the inhibitor of the complement system is an inhibitor of the complement system in humans. Complement inhibitors are grouped into two categories: soluble inhibitors and membrane- bound inhibitors. Preferably, the inhibitor of the complement system is a soluble complement inhibitor. Soluble complement inhibitors include C1 inhibitor (C1INH), complement factor I (CFI), complement factor H (CFH), complement factor H-like protein 1 (FHL-1), C4 binding protein (C4BP), clusterin and vitronectin. Membrane-bound regulators include CD46, CD55, CD59, CD35 and CUB and Sushi multiple domain 1 (CSMD1). The inhibitor of the complement system may be selected from: CFI, CFH, FHL-1, C1INH, C4BP, CD46, CD55, CD59, CD35, vitronectin, clusterin, and CSMD1, or fragments and/or variants thereof. Preferably, the inhibitor of the complement system is selected from: CFI, CFH, and FHL-1, or fragments and/or variants thereof. CFI The transgene may encode CFI, or a fragment and/or variant thereof. Complement factor I (CFI) is a trypsin-like serine protease that inhibits the complement system by cleaving three peptide bonds in the alpha-chain of C3b and two bonds in the alpha-chain of C4b thereby inactivating these proteins. CFI is a glycoprotein heterodimer consisting of a disulfide linked heavy chain and light chain. The heavy chain has four domains: an FI membrane attack complex (FIMAC) domain, CD5 domain, and low-density lipoprotein receptor 1 and 2 (LDLr1 and LDLr2) domains. The heavy chain plays an inhibitory role in maintaining the enzyme inactive until it meets the complex formed by the substrate (either C3b or C4b) and a cofactor protein (Factor H, C4b-binding protein, complement receptor 1, and membrane cofactor protein). Upon binding of the enzyme to the substrate:cofactor complex, the heavy:light chain interface is disrupted, and the enzyme activated by allostery. The light chain contains only the serine protease domain. This domain contains the catalytic triad His-362, Asp-411, and Ser-507, which is responsible for specific cleavage of C3b and C4b. The CFI or a fragment and/or variant thereof may be capable of cleaving C3b into iC3b and/or may be capable of cleaving iC3b into C3d,g. The fragment and/or variant of CFI may retain at least 50%, at least 60%, at least 70%, at least 80%, at least 90%, at least 95% or 100% of the C3b-inactivating and iC3b-degradation activity of native CFI. The C3b-inactivating and iC3b-degradation activity of the fragment and/or variant of CFI and native CFI, may be determined using any suitable method known to those of skill in the art. For example, using a proteolytic assay. Preferably, the CFI is a human CFI. An example human CFI is the CFI having the UniProtKB accession number P05156. Suitably, the CFI may comprise or consist of the polypeptide sequence shown as SEQ ID NO: 13, or a variant which is at least 70% identical to SEQ ID NO: 13. Illustrative CFI polypeptide sequence (SEQ ID NO: 13): MKLLHVFLLFLCFHLRFCKVTYTSQEDLVEKKCLAKKYTHLSCDKVFCQPWQRCIEGTCVCKLPYQCP KNGTAVCATNRRSFPTYCQQKSLECLHPGTKFLNNGTCTAEGKFSVSLKHGNTDSEGIVEVKLVDQDK TMFICKSSWSMREANVACLDLGFQQGADTQRRFKLSDLSINSTECLHVHCRGLETSLAECTFTKRRTM GYQDFADVVCYTQKADSPMDDFFQCVNGKYISQMKACDGINDCGDQSDELCCKACQGKGFHCKSGVCI PSQYQCNGEVDCITGEDEVGCAGFASVTQEETEILTADMDAERRRIKSLLPKLSCGVKNRMHIRRKRI VGGKRAQLGDLPWQVAIKDASGITCGGIYIGGCWILTAAHCLRASKTHRYQIWTTVVDWIHPDLKRIV IEYVDRIIFHENYNAGTYQNDIALIEMKKDGNKKDCELPRSIPACVPWSPYLFQPNDTCIVSGWGREK DNERVFSLQWGEVKLISNCSKFYGNRFYEKEMECAGTYDGSIDACKGDSGGPLVCMDANNVTYVWGVV SWGENCGKPEFPGVYTKVANYFDWISYHVGRPFISQYNV Suitably, the variant may be at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99% identical to SEQ ID NO: 13. An example nucleotide sequence encoding CFI is NM_000204.5. Suitably, a transgene encoding CFI may comprise or consist of the polynucleotide sequence shown as SEQ ID NO: 14, or a variant which is at least 70% identical to SEQ ID NO: 14. Illustrative CFI polynucleotide sequence (SEQ ID NO: 14): ATGAAGCTTCTTCATGTTTTCCTGTTATTTCTGTGCTTCCACTTAAGGTTTTGCAAGGTCACTTATAC ATCTCAAGAGGATCTGGTGGAGAAAAAGTGCTTAGCAAAAAAATATACTCACCTCTCCTGCGATAAAG TCTTCTGCCAGCCATGGCAGAGATGCATTGAGGGCACCTGTGTTTGTAAACTACCGTATCAGTGCCCA AAGAATGGCACTGCAGTGTGTGCAACTAACAGGAGAAGCTTCCCAACATACTGTCAACAAAAGAGTTT GGAATGTCTTCATCCAGGGACAAAGTTTTTAAATAACGGAACATGCACAGCCGAAGGAAAGTTTAGTG TTTCCTTGAAGCATGGAAATACAGATTCAGAGGGAATAGTTGAAGTAAAACTTGTGGACCAAGATAAG ACAATGTTCATATGCAAAAGCAGCTGGAGCATGAGGGAAGCCAACGTGGCCTGCCTTGACCTTGGGTT TCAACAAGGTGCTGATACTCAAAGAAGGTTTAAGTTGTCTGATCTCTCTATAAATTCCACTGAATGTC TACATGTGCATTGCCGAGGATTAGAGACCAGTTTGGCTGAATGTACTTTTACTAAGAGAAGAACTATG GGTTACCAGGATTTCGCTGATGTGGTTTGTTATACACAGAAAGCAGATTCTCCAATGGATGACTTCTT TCAGTGTGTGAATGGGAAATACATTTCTCAGATGAAAGCCTGTGATGGTATCAATGATTGTGGAGACC AAAGTGATGAACTGTGTTGTAAAGCATGCCAAGGCAAAGGCTTCCATTGCAAATCGGGTGTTTGCATT CCAAGCCAGTATCAATGCAATGGTGAGGTGGACTGCATTACAGGGGAAGATGAAGTTGGCTGTGCAGG CTTTGCATCTGTGACTCAAGAAGAAACAGAAATTTTGACTGCTGACATGGATGCAGAAAGAAGACGGA TAAAATCATTATTACCTAAACTATCTTGTGGAGTTAAAAACAGAATGCACATTCGAAGGAAACGAATT GTGGGAGGAAAGCGAGCACAACTGGGAGACCTCCCATGGCAGGTGGCAATTAAGGATGCCAGTGGAAT CACCTGTGGGGGAATTTATATTGGTGGCTGTTGGATTCTGACTGCTGCACATTGTCTCAGAGCCAGTA AAACTCATCGTTACCAAATATGGACAACAGTAGTAGACTGGATACACCCCGACCTTAAACGTATAGTA ATTGAATACGTGGATAGAATTATTTTCCATGAAAACTACAATGCAGGCACTTACCAAAATGACATCGC TTTGATTGAAATGAAAAAAGACGGAAACAAAAAAGATTGTGAGCTGCCTCGTTCCATCCCTGCCTGTG TCCCCTGGTCTCCTTACCTATTCCAACCTAATGATACATGCATCGTTTCTGGCTGGGGACGAGAAAAA GATAACGAAAGAGTCTTTTCACTTCAGTGGGGTGAAGTTAAACTAATAAGCAACTGCTCTAAGTTTTA CGGAAATCGTTTCTATGAAAAAGAAATGGAATGTGCAGGTACATATGATGGTTCCATCGATGCCTGTA AAGGGGACTCTGGAGGCCCCTTAGTCTGTATGGATGCCAACAATGTGACTTATGTCTGGGGTGTTGTG AGTTGGGGGGAAAACTGTGGAAAACCAGAGTTCCCAGGTGTTTACACCAAAGTGGCCAATTATTTTGA CTGGATTAGCTACCATGTAGGAAGGCCTTTTATTTCTCAGTACAATGTATAA Suitably, the variant may be at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99% identical to SEQ ID NO: 14. CFH The transgene may encode CFH, or a fragment and/or variant thereof. Complement factor H (CFH) regulates complement activation on self-cells and surfaces. CFH competes for binding of complement factor B (CFB) to C3b, acts as a cofactor for CFI- catalysed proteolytic cleavage of C3b, and accelerates the irreversible dissociation of C3bBb and C3b2Bb into their separate components. Thus, CFH not only inhibits formation of the convertases but it also shortens the lifespan of any convertase complex that forms. CFH is a large (155 kDa) soluble glycoprotein. CFH is composed from a total of 20 domains, each containing approximately 60 amino acid residues and termed complement control protein modules (CCPs) or short consensus repeats that are joined by short linkers consisting of 3–8 residues. The CCP modules are numbered from 1–20 (from the N-terminus of the protein): CCPs 1–4 and CCPs 19–20 engage with C3b while CCPs 7 and CCPs 19–20 bind to GAGs and sialic acid. The CFH or a fragment and/or variant thereof may be capable of binding C3b and/or C3d; and/or acting as a cofactor for the CFI-catalysed proteolytic cleavage of C3b; and/or increasing the irreversible dissociation of C3bBb and C3b2Bb into their separate components. The fragment and/or variant of CFH may retain at least 50%, at least 60%, at least 70%, at least 80%, at least 90%, at least 95% or 100% of the activity of native CFH. The activity of the fragment and/or variant of CFH and native CFH may be determined using any suitable method known to those of skill in the art. Preferably, the CFH is a human CFH. An example human CFH is the CFH having the UniProtKB accession number P08603. Suitably, the CFH may comprise or consist of the polypeptide sequence shown as SEQ ID NO: 15, or a variant which is at least 70% identical to SEQ ID NO: 15. Illustrative CFH polypeptide sequence (SEQ ID NO: 15): MRLLAKIICLMLWAICVAEDCNELPPRRNTEILTGSWSDQTYPEGTQAIYKCRPGYRSLGNVIMVCRK GEWVALNPLRKCQKRPCGHPGDTPFGTFTLTGGNVFEYGVKAVYTCNEGYQLLGEINYRECDTDGWTN DIPICEVVKCLPVTAPENGKIVSSAMEPDREYHFGQAVRFVCNSGYKIEGDEEMHCSDDGFWSKEKPK CVEISCKSPDVINGSPISQKIIYKENERFQYKCNMGYEYSERGDAVCTESGWRPLPSCEEKSCDNPYI PNGDYSPLRIKHRTGDEITYQCRNGFYPATRGNTAKCTSTGWIPAPRCTLKPCDYPDIKHGGLYHENM RRPYFPVAVGKYYSYYCDEHFETPSGSYWDHIHCTQDGWSPAVPCLRKCYFPYLENGYNQNYGRKFVQ GKSIDVACHPGYALPKAQTTVTCMENGWSPTPRCIRVKTCSKSSIDIENGFISESQYTYALKEKAKYQ CKLGYVTADGETSGSITCGKDGWSAQPTCIKSCDIPVFMNARTKNDFTWFKLNDTLDYECHDGYESNT GSTTGSIVCGYNGWSDLPICYERECELPKIDVHLVPDRKKDQYKVGEVLKFSCKPGFTIVGPNSVQCY HFGLSPDLPICKEQVQSCGPPPELLNGNVKEKTKEEYGHSEVVEYYCNPRFLMKGPNKIQCVDGEWTT LPVCIVEESTCGDIPELEHGWAQLSSPPYYYGDSVEFNCSESFTMIGHRSITCIHGVWTQLPQCVAID KLKKCKSSNLIILEEHLKNKKEFDHNSNIRYRCRGKEGWIHTVCINGRWDPEVNCSMAQIQLCPPPPQ IPNSHNMTTTLNYRDGEKVSVLCQENYLIQEGEEITCKDGRWQSIPLCVEKIPCSQPPQIEHGTINSS RSSQESYAHGTKLSYTCEGGFRISEENETTCYMGKWSSPPQCEGLPCKSPPEISHGVVAHMSDSYQYG EEVTYKCFEGFGIDGPAIAKCLGEKWSHPPSCIKTDCLSLPSFENAIPMGEKKDVYKAGEQVTYTCAT YYKMDGASNVTCINSRWTGRPTCRDTSCVNPPTVQNAYIVSRQMSKYPSGERVRYQCRSPYEMFGDEE VMCLNGNWTEPPQCKDSTGKCGPPPPIDNGDITSFPLSVYAPASSVEYQCQNLYQLEGNKRITCRNGQ WSEPPKCLHPCVISREIMENYNIALRWTAKQKLYSRTGESVEFVCKRGYRLSSRSHTLRTTCWDGKLE YPTCAKR Suitably, the variant may be at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99% identical to SEQ ID NO: 15. An example nucleotide sequence encoding CFH is NM_000186.4. Suitably, a transgene encoding CFH may comprise or consist of the polynucleotide sequence shown as SEQ ID NO: 16, or a variant which is at least 70% identical to SEQ ID NO: 16. Illustrative CFH polynucleotide sequence (SEQ ID NO: 16): ATGAGACTTCTAGCAAAGATTATTTGCCTTATGTTATGGGCTATTTGTGTAGCAGAAGATTGCAATGA ACTTCCTCCAAGAAGAAATACAGAAATTCTGACAGGTTCCTGGTCTGACCAAACATATCCAGAAGGCA CCCAGGCTATCTATAAATGCCGCCCTGGATATAGATCTCTTGGAAATGTAATAATGGTATGCAGGAAG GGAGAATGGGTTGCTCTTAATCCATTAAGGAAATGTCAGAAAAGGCCCTGTGGACATCCTGGAGATAC TCCTTTTGGTACTTTTACCCTTACAGGAGGAAATGTGTTTGAATATGGTGTAAAAGCTGTGTATACAT GTAATGAGGGGTATCAATTGCTAGGTGAGATTAATTACCGTGAATGTGACACAGATGGATGGACCAAT GATATTCCTATATGTGAAGTTGTGAAGTGTTTACCAGTGACAGCACCAGAGAATGGAAAAATTGTCAG TAGTGCAATGGAACCAGATCGGGAATACCATTTTGGACAAGCAGTACGGTTTGTATGTAACTCAGGCT ACAAGATTGAAGGAGATGAAGAAATGCATTGTTCAGACGATGGTTTTTGGAGTAAAGAGAAACCAAAG TGTGTGGAAATTTCATGCAAATCCCCAGATGTTATAAATGGATCTCCTATATCTCAGAAGATTATTTA TAAGGAGAATGAACGATTTCAATATAAATGTAACATGGGTTATGAATACAGTGAAAGAGGAGATGCTG TATGCACTGAATCTGGATGGCGTCCGTTGCCTTCATGTGAAGAAAAATCATGTGATAATCCTTATATT CCAAATGGTGACTACTCACCTTTAAGGATTAAACACAGAACTGGAGATGAAATCACGTACCAGTGTAG AAATGGTTTTTATCCTGCAACCCGGGGAAATACAGCAAAATGCACAAGTACTGGCTGGATACCTGCTC CGAGATGTACCTTGAAACCTTGTGATTATCCAGACATTAAACATGGAGGTCTATATCATGAGAATATG CGTAGACCATACTTTCCAGTAGCTGTAGGAAAATATTACTCCTATTACTGTGATGAACATTTTGAGAC TCCGTCAGGAAGTTACTGGGATCACATTCATTGCACACAAGATGGATGGTCGCCAGCAGTACCATGCC TCAGAAAATGTTATTTTCCTTATTTGGAAAATGGATATAATCAAAATCATGGAAGAAAGTTTGTACAG GGTAAATCTATAGACGTTGCCTGCCATCCTGGCTACGCTCTTCCAAAAGCGCAGACCACAGTTACATG TATGGAGAATGGCTGGTCTCCTACTCCCAGATGCATCCGTGTCAAAACATGTTCCAAATCAAGTATAG ATATTGAGAATGGGTTTATTTCTGAATCTCAGTATACATATGCCTTAAAAGAAAAAGCGAAATATCAA TGCAAACTAGGATATGTAACAGCAGATGGTGAAACATCAGGATCAATTACATGTGGGAAAGATGGATG GTCAGCTCAACCCACGTGCATTAAATCTTGTGATATCCCAGTATTTATGAATGCCAGAACTAAAAATG ACTTCACATGGTTTAAGCTGAATGACACATTGGACTATGAATGCCATGATGGTTATGAAAGCAATACT GGAAGCACCACTGGTTCCATAGTGTGTGGTTACAATGGTTGGTCTGATTTACCCATATGTTATGAAAG AGAATGCGAACTTCCTAAAATAGATGTACACTTAGTTCCTGATCGCAAGAAAGACCAGTATAAAGTTG GAGAGGTGTTGAAATTCTCCTGCAAACCAGGATTTACAATAGTTGGACCTAATTCCGTTCAGTGCTAC CACTTTGGATTGTCTCCTGACCTCCCAATATGTAAAGAGCAAGTACAATCATGTGGTCCACCTCCTGA ACTCCTCAATGGGAATGTTAAGGAAAAAACGAAAGAAGAATATGGACACAGTGAAGTGGTGGAATATT ATTGCAATCCTAGATTTCTAATGAAGGGACCTAATAAAATTCAATGTGTTGATGGAGAGTGGACAACT TTACCAGTGTGTATTGTGGAGGAGAGTACCTGTGGAGATATACCTGAACTTGAACATGGCTGGGCCCA GCTTTCTTCCCCTCCTTATTACTATGGAGATTCAGTGGAATTCAATTGCTCAGAATCATTTACAATGA TTGGACACAGATCAATTACGTGTATTCATGGAGTATGGACCCAACTTCCCCAGTGTGTGGCAATAGAT AAACTTAAGAAGTGCAAATCATCAAATTTAATTATACTTGAGGAACATTTAAAAAACAAGAAGGAATT CGATCATAATTCTAACATAAGGTACAGATGTAGAGGAAAAGAAGGATGGATACACACAGTCTGCATAA ATGGAAGATGGGATCCAGAAGTGAACTGCTCAATGGCACAAATACAATTATGCCCACCTCCACCTCAG ATTCCCAATTCTCACAATATGACAACCACACTGAATTATCGGGATGGAGAAAAAGTATCTGTTCTTTG CCAAGAAAATTATCTAATTCAGGAAGGAGAAGAAATTACATGCAAAGATGGAAGATGGCAGTCAATAC CACTCTGTGTTGAAAAAATTCCATGTTCACAACCACCTCAGATAGAACACGGAACCATTAATTCATCC AGGTCTTCACAAGAAAGTTATGCACATGGGACTAAATTGAGTTATACTTGTGAGGGTGGTTTCAGGAT ATCTGAAGAAAATGAAACAACATGCTACATGGGAAAATGGAGTTCTCCACCTCAGTGTGAAGGCCTTC CTTGTAAATCTCCACCTGAGATTTCTCATGGTGTTGTAGCTCACATGTCAGACAGTTATCAGTATGGA GAAGAAGTTACGTACAAATGTTTTGAAGGTTTTGGAATTGATGGGCCTGCAATTGCAAAATGCTTAGG AGAAAAATGGTCTCACCCTCCATCATGCATAAAAACAGATTGTCTCAGTTTACCTAGCTTTGAAAATG CCATACCCATGGGAGAGAAGAAGGATGTGTATAAGGCGGGTGAGCAAGTGACTTACACTTGTGCAACA TATTACAAAATGGATGGAGCCAGTAATGTAACATGCATTAATAGCAGATGGACAGGAAGGCCAACATG CAGAGACACCTCCTGTGTGAATCCGCCCACAGTACAAAATGCTTATATAGTGTCGAGACAGATGAGTA AATATCCATCTGGTGAGAGAGTACGTTATCAATGTAGGAGCCCTTATGAAATGTTTGGGGATGAAGAA GTGATGTGTTTAAATGGAAACTGGACGGAACCACCTCAATGCAAAGATTCTACAGGAAAATGTGGGCC CCCTCCACCTATTGACAATGGGGACATTACTTCATTCCCGTTGTCAGTATATGCTCCAGCTTCATCAG TTGAGTACCAATGCCAGAACTTGTATCAACTTGAGGGTAACAAGCGAATAACATGTAGAAATGGACAA TGGTCAGAACCACCAAAATGCTTACATCCGTGTGTAATATCCCGAGAAATTATGGAAAATTATAACAT AGCATTAAGGTGGACAGCCAAACAGAAGCTTTATTCGAGAACAGGTGAATCAGTTGAATTTGTGTGTA AACGGGGATATCGTCTTTCATCACGTTCTCACACATTGCGAACAACATGTTGGGATGGGAAACTGGAG TATCCAACTTGTGCAAAAAGATAG Suitably, the variant may be at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99% identical to SEQ ID NO: 16. The CFH fragment may be a splice variant. For example, complement factor H-like protein 1 (FHL-1) is a CFH gene splice variant, which is almost identical to the N-terminal 7 domains of CFH (CCPs 1-7). FHL-1 The transgene may encode FHL-1, or a fragment and/or variant thereof. FHL-1 or a fragment and/or variant thereof may be capable of binding C3b and/or C3d. The fragment and/or variant of FHL-1 may retain at least 50%, at least 60%, at least 70%, at least 80%, at least 90%, at least 95% or 100% of the activity of native FHL-1. The activity of the fragment and/or variant of FHL-1 and native FHL-1 may be determined using any suitable method known to those of skill in the art. Preferably, the FHL-1 is a human FHL-1. An example human FHL-1 is the FHL-1 having the NCBI Reference Sequence: NP_001014975.1. Suitably, the FHL-1 may comprise or consist of the polypeptide sequence shown as SEQ ID NO: 17, or a variant which is at least 70% identical to SEQ ID NO: 17. Illustrative FHL-1 polypeptide sequence (SEQ ID NO: 17): MRLLAKIICLMLWAICVAEDCNELPPRRNTEILTGSWSDQTYPEGTQAIYKCRPGYRSLGNVIMVCRK GEWVALNPLRKCQKRPCGHPGDTPFGTFTLTGGNVFEYGVKAVYTCNEGYQLLGEINYRECDTDGWTN DIPICEVVKCLPVTAPENGKIVSSAMEPDREYHFGQAVRFVCNSGYKIEGDEEMHCSDDGFWSKEKPK CVEISCKSPDVINGSPISQKIIYKENERFQYKCNMGYEYSERGDAVCTESGWRPLPSCEEKSCDNPYI PNGDYSPLRIKHRTGDEITYQCRNGFYPATRGNTAKCTSTGWIPAPRCTLKPCDYPDIKHGGLYHENM RRPYFPVAVGKYYSYYCDEHFETPSGSYWDHIHCTQDGWSPAVPCLRKCYFPYLENGYNQNHGRKFVQ GKSIDVACHPGYALPKAQTTVTCMENGWSPTPRCIRVSFTL Suitably, the variant may be at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99% identical to SEQ ID NO: 17. An example nucleotide sequence encoding FHL-1 is NM_001014975.2. Suitably, a transgene encoding FHL-1 may comprise or consist of the polynucleotide sequence shown as SEQ ID NO: 18, or a variant which is at least 70% identical to SEQ ID NO: 18. Illustrative FHL-1 polynucleotide sequence (SEQ ID NO: 18): ATGAGACTTCTAGCAAAGATTATTTGCCTTATGTTATGGGCTATTTGTGTAGCAGAAGATTGCAATGA ACTTCCTCCAAGAAGAAATACAGAAATTCTGACAGGTTCCTGGTCTGACCAAACATATCCAGAAGGCA CCCAGGCTATCTATAAATGCCGCCCTGGATATAGATCTCTTGGAAATGTAATAATGGTATGCAGGAAG GGAGAATGGGTTGCTCTTAATCCATTAAGGAAATGTCAGAAAAGGCCCTGTGGACATCCTGGAGATAC TCCTTTTGGTACTTTTACCCTTACAGGAGGAAATGTGTTTGAATATGGTGTAAAAGCTGTGTATACAT GTAATGAGGGGTATCAATTGCTAGGTGAGATTAATTACCGTGAATGTGACACAGATGGATGGACCAAT GATATTCCTATATGTGAAGTTGTGAAGTGTTTACCAGTGACAGCACCAGAGAATGGAAAAATTGTCAG TAGTGCAATGGAACCAGATCGGGAATACCATTTTGGACAAGCAGTACGGTTTGTATGTAACTCAGGCT ACAAGATTGAAGGAGATGAAGAAATGCATTGTTCAGACGATGGTTTTTGGAGTAAAGAGAAACCAAAG TGTGTGGAAATTTCATGCAAATCCCCAGATGTTATAAATGGATCTCCTATATCTCAGAAGATTATTTA TAAGGAGAATGAACGATTTCAATATAAATGTAACATGGGTTATGAATACAGTGAAAGAGGAGATGCTG TATGCACTGAATCTGGATGGCGTCCGTTGCCTTCATGTGAAGAAAAATCATGTGATAATCCTTATATT CCAAATGGTGACTACTCACCTTTAAGGATTAAACACAGAACTGGAGATGAAATCACGTACCAGTGTAG AAATGGTTTTTATCCTGCAACCCGGGGAAATACAGCAAAATGCACAAGTACTGGCTGGATACCTGCTC CGAGATGTACCTTGAAACCTTGTGATTATCCAGACATTAAACATGGAGGTCTATATCATGAGAATATG CGTAGACCATACTTTCCAGTAGCTGTAGGAAAATATTACTCCTATTACTGTGATGAACATTTTGAGAC TCCGTCAGGAAGTTACTGGGATCACATTCATTGCACACAAGATGGATGGTCGCCAGCAGTACCATGCC TCAGAAAATGTTATTTTCCTTATTTGGAAAATGGATATAATCAAAATCATGGAAGAAAGTTTGTACAG GGTAAATCTATAGACGTTGCCTGCCATCCTGGCTACGCTCTTCCAAAAGCGCAGACCACAGTTACATG TATGGAGAATGGCTGGTCTCCTACTCCCAGATGCATCCGTGTCAGCTTTACCCTCTGA Suitably, the variant may be at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99% identical to SEQ ID NO: 18. In one embodiment, the polynucleotide or vector does not comprise a nephrotic syndrome- associated transgene, such as NPHS2, ADCK4, ALG1, ARHGAP24, ARGHDIA, CD151, CD2AP, COQ2, COQ6, DGKE, E2F3, EMP2, KANK2, LAGE3, LMNA, LMX1B, MAFB, NUP85, NUP93, NXF5, OSGEP, PAX2, PDSS2, PMM2, PODXL, SCARB2, SGPL1, Smad7, TP53RK, TPRKB, VDR, WDR73, WT1, ZMPSTE24, APOL1, NPHS1, TRPC6, NUP107, NUP133, NUP160, ACTN4, INF2, ANKFY1, ANLN, CRB2, ITGA3, KANK1, KANK4, MAGI2, MYO1E, OCRL, PTPRO, SMARCAL1, SYNPO, TBC1D8B, XPO5, TNS2 or NLRP3. In one embodiment, the polynucleotide or vector does not comprise a vascular endothelial growth factor (VEGFC) transgene. In one embodiment, the polynucleotide or vector does not comprise a COL4A3, COL4A4, or COL4A5 transgene. In one embodiment, the polynucleotide or vector does not comprise a nucleotide sequence encoding a complement protein, such as CFI, CFH, FHL-1, C1INH, C4BP, MASP2, C3, C5aR1, C5, C5a, CD55, CD35, CD46, CD59, vitronectin, or clusterin. In one embodiment, the polynucleotide or vector does not comprise a NPHS2, ADCK4, ALG1, ARHGAP24, ARGHDIA, CD151, CD2AP, COQ2, COQ6, DGKE, E2F3, EMP2, KANK2, LAGE3, LMNA, LMX1B, MAFB, NUP85, NUP93, NXF5, OSGEP, PAX2, PDSS2, PMM2, PODXL, SCARB2, SGPL1, Smad7, TP53RK, TPRKB, VDR, WDR73, WT1, ZMPSTE24, APOL1, NPHS1, TRPC6, NUP107, NUP133, NUP160, ACTN4, INF2, ANKFY1, ANLN, CRB2, ITGA3, KANK1, KANK4, MAGI2, MYO1E, OCRL, PTPRO, SMARCAL1, SYNPO, TBC1D8B, XPO5, TNS2. NLRP3, VEGFC, COL4A3, COL4A4, COL4A5, CFI, CFH, FHL-1, C1INH, C4BP, MASP2, C3, C5aR1, C5, C5a, CD55, CD35, CD46, CD59, vitronectin, or clusterin transgene. Other regulatory elements In addition to the promoter referred to herein, the polynucleotide or vector may comprise one or more further regulatory sequences which may act pre- or post-transcriptionally. Suitably, the transgene may be operably linked to one or more further regulatory sequences. The one or more further regulatory sequences may facilitate expression of the encoded polypeptide in glomerular cells (e.g. podocytes). “Regulatory sequences” are any sequences which facilitate expression of the polypeptides, e.g. act to increase expression of a transcript or to enhance mRNA stability. Suitable further regulatory sequences include for example enhancer elements, post-transcriptional regulatory elements and polyadenylation sites. Enhancers The polynucleotide or vector of the invention may comprise an enhancer. Suitably, the enhancer may be operably linked to the transgene. The enhancer may facilitate expression of the transgene in glomerular cells (e.g. podocytes). Suitably, the enhancer is a mammalian enhancer e.g. a human enhancer. An “enhancer” is a region of DNA that can be bound by proteins (activators) to increase the likelihood that transcription of a particular gene will occur. Enhancers are cis-acting. They can be located up to 1 Mbp (1,000,000 bp) away from the gene, upstream or downstream from the start site. Any suitable enhancer may be used, the selection of which may be readily made by the skilled person. The polynucleotide or vector defined herein may comprise a tissue specific enhancer. As used herein, a “tissue-specific enhancer” is an enhancer which preferentially facilitates expression of a gene in specific cells or tissues. Suitably, a tissue-specific enhancer may facilitate higher expression of a gene in specific cells-types as compared to other cell-types. Higher expression may be measured for example by measuring the expression of a transgene, e.g. GFP, operably linked to the enhancer, wherein expression of the transgene correlates with the ability of the enhancer to facilitate expression of a gene. For example, a tissue-specific enhancer may be an enhancer which facilitates gene expression levels at least 10% higher, at least 20% higher, at least 30% higher, at least 40% higher, at least 50% higher, at least 100% higher, at least 200% higher, at least 300% higher, at least 400% higher, at least 500% higher, or at least 1000% higher in a specific cell-type compared to expression levels in other cell types. Suitable tissue-specific enhancer will be well known to those of skill in the art. The enhancer may be a kidney-specific enhancer, preferably a glomerular-specific enhancer, more preferably a podocyte-specific enhancer. Suitably, the enhancer may be operably linked to the transgene. Suitably, the enhancer may be or may be derived from an enhancer associated with a gene with selective expression in human kidney, glomerular cells, and/or podocytes. Methods to identify the enhancer regions associated with genes will be well known to those of skill in the art. Suitably, the podocyte-specific enhancer may be a NPHS1 or a NPHS2 enhancer, or a fragment or derivative thereof. Suitably, the podocyte-specific enhancer may be a NPHS1 enhancer, or a fragment or derivative thereof. A NPHS1 enhancer has been described in Guo, G., et al., 2004. Journal of the American Society of Nephrology, 15(11), pp.2851-2856. A 186-bp fragment from the human NPHS1 promoter was capable of directing podocyte-specific expression of a β-galactosidase transgene when placed in front of a heterologous minimal promoter in transgenic mice. Suitably, the NPHS1 enhancer may comprise or consist of the nucleotide sequence shown as SEQ ID NO: 19, or a variant which is at least 70% identical to SEQ ID NO: 19. Exemplary NPHS1 enhancer (SEQ ID NO: 19): ctgctgagctgggagaccaccttgatctgacttctcccatcttcccagcctaagccaggccctggggt cacggaggctggggaggcaccgaggaacgcgcctggcatgtgctgacaggggattttatgctccagct gggccagctgggaggagcctgctgggcagaggccagagctgggggctctg Suitably, the variant may be at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 19. Kozak sequence The polynucleotide or vector may comprise a Kozak sequence. Suitably, the Kozak sequence may be operably linked to the transgene. A Kozak sequence may be inserted before the start codon of the protein to improve the initiation of translation. Suitable Kozak sequences will be well known to those of skill in the art. Suitably, the Kozak sequence may comprise or consist of the nucleotide sequence shown as SEQ ID NO: 20, or a variant which is at least 65% identical to SEQ ID NO: 20. Exemplary Kozak sequence (SEQ ID NO: 20): GCCGCCACCAUGG Suitably, the variant may be at least 75%, at least 85%, or at least 90% identical to SEQ ID NO: 20. Post-transcriptional regulatory elements The polynucleotide or vector defined herein may comprise a post-transcriptional regulatory element. Suitably, the post-transcriptional regulatory element may be operably linked to the transgene. The post-transcriptional regulatory element may improve gene expression. The polynucleotide may comprise a Woodchuck Hepatitis Virus Post-transcriptional Regulatory Element (WPRE). Suitably, the WPRE may be operably linked to the transgene. The WPRE sequence may have substitutions, deletions, or insertions within the X-antigen promoter and/or the initiation codon of the X-antigen. This may prevent the production of a functional X-antigen. Suitably, the WPRE may comprise or consist of the nucleotide sequence shown as SEQ ID NO: 21, or a variant which is at least 70% identical to SEQ ID NO: 21. Exemplary WPRE (SEQ ID NO: 21): aatcaacctctggattacaaaatttgtgaaagattgactggtattcttaactatgttgctccttttac gctatgtggatacgctgctttaatgcctttgtatcatgctattgcttcccgtatggctttcattttct cctccttgtataaatcctggttgctgtctctttatgaggagttgtggcccgttgtcaggcaacgtggc gtggtgtgcactgtgtttgctgacgcaacccccactggttggggcattgccaccacctgtcagctcct ttccgggactttcgctttccccctccctattgccacggcggaactcatcgccgcctgccttgcccgct gctggacaggggctcggctgttgggcactgacaattccgtggtgttgtcggggaaatcatcgtccttt ccttggctgctcgcctgtgttgccacctggattctgcgcgggacgtccttctgctacgtcccttcggc cctcaatccagcggaccttccttcccgcggcctgctgccggctctgcggcctcttccgcgtcttcgcc ttcgccctcagacgagtcggatctccctttgggccgcctccccgc Suitably, the variant may be at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99% identical to SEQ ID NO: 21. Polyadenylation signal The polynucleotide or vector defined herein may comprise a polyadenylation signal. Suitably, the polyadenylation signal may be operably linked to the transgene. The polyadenylation signal may improve gene expression. Suitable polyadenylation signals include the early SV40 polyadenylation signal (SV40pA), a chicken beta-globin polyadenylation signal, a bovine growth hormone polyadenylation signal (bGH), or a soluble neuropilin-1 polyadenylation signal. In some embodiments, the polyadenylation signal is an early SV40 polyadenylation signal (SV40pA) or a chicken beta- globin polyadenylation signal. Preferably, the polyadenylation signal is an early SV40 polyadenylation signal (SV40pA). Suitably, the polyadenylation signal may comprise or consist of the nucleotide sequence shown as SEQ ID NO: 22, or a variant which is at least 70% identical to SEQ ID NO: 22. Suitably, the variant may be at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99% identical to SEQ ID NO: 22. Exemplary SV40pA signal sequence (SEQ ID NO: 22): aacttgtttattgcagcttataatggttacaaataaagcaatagcatcacaaatttcacaaataaagc atttttttcactgcattctagttgtggtttgtccaaactcatcaatgtatcttatcatgtctggatc Suitably, the polyadenylation signal may comprise or consist of the nucleotide sequence shown as SEQ ID NO: 23, or a variant which is at least 70% identical to SEQ ID NO: 23. Suitably, the variant may be at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99% identical to SEQ ID NO: 23. Exemplary bGH poly(A) signal sequence (SEQ ID NO: 23): ctgtgccttctagttgccagccatctgttgtttgcccctcccccgtgccttccttgaccctggaaggt gccactcccactgtcctttcctaataaaatgaggaaattgcatcgcattgtctgagtaggtgtcattc tattctggggggtggggtggggcaggacagcaagggggaggattgggaagacaatagcaggcatgctg gggatgcggtgggctctatgg Suitably, the polyadenylation signal may comprise or consist of the nucleotide sequence shown as SEQ ID NO: 24, or a variant which is at least 70% identical to SEQ ID NO: 24. Suitably, the variant may be at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99% identical to SEQ ID NO: 24. Exemplary soluble neuropilin-1 polyadenylation signal (SEQ ID NO: 24): aaataaaatacgaaatg Suitably, the polyadenylation signal may comprise or consist of the nucleotide sequence shown as SEQ ID NO: 25, or a variant which is at least 70% identical to SEQ ID NO: 25. Suitably, the variant may be at least 75%, at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99% identical to SEQ ID NO: 25. Exemplary chicken beta-globin polyadenylation signal (SEQ ID NO: 25) Caataaaagatctttattttcattagatctgtgtgttggttttttgtgtg Vectors In one aspect, the present invention provides a vector comprising the promoter or polynucleotide described herein. A vector is a tool that allows or facilitates the transfer of an entity from one environment to another. The four major types of vectors are plasmids, viral vectors, cosmids, and artificial chromosomes. Preferably, the vector of the present invention is a viral vector. The vector of the invention is preferably an adeno-associated viral (AAV) vector, although it is contemplated that other viral vectors may be used. Other viral vectors include retroviral vectors, lentiviral vectors, adenoviral vectors, herpes simplex viral vectors, alphaviral vectors, flaviviral vectors, rhabdoviral vectors, measles viral vector, Newcastle disease viral vectors, poxviral vectors, and picornaviral vectors (see e.g. Lundstrom, K., 2018. Diseases, 6(2), p.42). The vector of the present invention may be in the form of a viral vector particle. Preferably, the viral vector of the present invention is in the form of an AAV vector particle. Methods of preparing and modifying viral vectors and viral vector particles, such as those derived from AAV, are well known in the art. Suitable methods are described in Ayuso, E., et al., 2010. Current gene therapy, 10(6), pp.423-436, Merten, O.W., et al., 2016. Molecular Therapy-Methods & Clinical Development, 3, p.16017; and Nadeau, I. and Kamen, A., 2003. Biotechnology advances, 20(7-8), pp.475-489. The vector of the present invention may be capable of transducing kidney cells. In some embodiments, the vector of the present invention is capable of specifically transducing kidney cells. The vector of the present invention is preferably capable of transducing glomerular cells (e.g. podocytes). In some embodiments, the vector of the present invention is capable of specifically transducing glomerular cells (e.g. podocytes). The vector of the present invention is preferably capable of transducing podocytes. In some embodiments, the vector of the present invention is capable of specifically transducing podocytes. Adeno-associated viral (AAV) vectors As described herein, the present invention provides an adeno-associated viral (AAV) comprising a transgene wherein the transgene is operably linked to a 5’-truncated podocin promoter. AAV genome The AAV vector or AAV vector particle may comprise an AAV genome or a fragment or derivative thereof. An AAV genome is a polynucleotide sequence, which may encode functions needed for production of an AAV particle. These functions include those operating in the replication and packaging cycle of AAV in a host cell, including encapsidation of the AAV genome into an AAV particle. Naturally occurring AAVs are replication-deficient and rely on the provision of helper functions in trans for completion of a replication and packaging cycle. Accordingly, the AAV genome of the AAV vector of the invention is typically replication-deficient. The AAV genome may be in single-stranded form (ssAAV), either positive or negative-sense, or alternatively in double-stranded form (dsAAV). The use of a double-stranded form allows bypass of the DNA replication step in the target cell and so can accelerate transgene expression. The maximum packaging capacity of the single-stranded form is larger than the double-stranded form. Suitably, the AAV genome is in single-stranded form. AAVs occurring in nature may be classified according to various biological systems. The AAV genome may be from any naturally derived serotype, isolate or clade of AAV. AAV may be referred to in terms of their serotype. A serotype corresponds to a variant subspecies of AAV which, owing to its profile of expression of capsid surface antigens, has a distinctive reactivity which can be used to distinguish it from other variant subspecies. Typically, an AAV vector particle having a particular AAV serotype does not efficiently cross- react with neutralising antibodies specific for any other AAV serotype. AAV serotypes include AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV10 and AAV11. AAV may also be referred to in terms of clades or clones. This refers to the phylogenetic relationship of naturally derived AAVs, and typically to a phylogenetic group of AAVs which can be traced back to a common ancestor, and includes all descendants thereof. Additionally, AAVs may be referred to in terms of a specific isolate, i.e. a genetic isolate of a specific AAV found in nature. The term genetic isolate describes a population of AAVs which has undergone limited genetic mixing with other naturally occurring AAVs, thereby defining a recognisably distinct population at a genetic level. Typically, the AAV genome of a naturally derived serotype, isolate or clade of AAV comprises at least one inverted terminal repeat sequence (ITR). An ITR sequence acts in cis to provide a functional origin of replication and allows for integration and excision of the vector from the genome of a cell. ITRs may be the only sequences required in cis next to the therapeutic gene. The AAV genome may also comprise packaging genes, such as rep and/or cap genes which encode packaging functions for an AAV particle. A promoter may be operably linked to each of the packaging genes. Specific examples of such promoters include the p5, p19 and p40 promoters. For example, the p5 and p19 promoters are generally used to express the rep gene, while the p40 promoter is generally used to express the cap gene. The rep gene encodes one or more of the proteins Rep78, Rep68, Rep52 and Rep40 or variants thereof. The cap gene encodes one or more capsid proteins such as VP1, VP2 and VP3 or variants thereof. These proteins make up the capsid of an AAV particle, which determines the AAV serotype. VP1, VP2, and VP3 may be produced by alternate mRNA splicing (Trempe, J.P. and Carter, B.J., 1988. Journal of virology, 62(9), pp.3356-3363). Thus, VP1, VP2 and VP3 may have identical sequences, but wherein VP2 is truncated at the N-terminus relative to VP1, and VP3 is truncated at the N-terminus relative to VP2. The AAV genome may be the full genome of a naturally occurring AAV. For example, a vector comprising a full AAV genome may be used to prepare an AAV vector or vector particle. Preferably, the AAV genome is derivatised for the purpose of administration to patients. Such derivatisation is standard in the art and the invention encompasses the use of any known derivative of an AAV genome, and derivatives which could be generated by applying techniques known in the art. The AAV genome may be a derivative of any naturally occurring AAV. Suitably, the AAV genome is a derivative of AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV10, or AAV11. Suitably, the AAV genome is a derivative of AAV2. Derivatives of an AAV genome include any truncated or modified forms of an AAV genome which allow for expression of a transgene from an AAV vector of the invention in vivo. Typically, it is possible to truncate the AAV genome significantly to include minimal viral sequence yet retain the above function. This is preferred for safety reasons to reduce the risk of recombination of the vector with wild-type virus, and also to avoid triggering a cellular immune response by the presence of viral gene proteins in the target cell. Typically, a derivative will include at least one inverted terminal repeat sequence (ITR), preferably more than one ITR, such as two ITRs or more. One or more of the ITRs may be derived from AAV genomes having different serotypes, or may be a chimeric or mutant ITR. A preferred mutant ITR is one having a deletion of a trs (terminal resolution site). This deletion allows for continued replication of the genome to generate a single-stranded genome which contains both coding and complementary sequences, i.e. a self-complementary AAV (scAAV) genome. This allows for bypass of DNA replication in the target cell, and so enables accelerated transgene expression. However, the maximum packaging capacity of a scAAV is reduced. Suitably, the AAV genome is not a scAAV genome. The AAV genome may comprise one or more ITR sequences from any naturally derived serotype, isolate or clade of AAV or a variant thereof. The AAV genome may comprise at least one, such as two, AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV10, or AAV11 ITRs, or variants thereof. Suitably, the AAV genome may comprise at least one, such as two, AAV2 ITRs. The inclusion of one or more ITRs is preferred to aid concatamer formation of the AAV vector in the nucleus of a host cell, for example following the conversion of single-stranded vector DNA into double-stranded DNA by the action of host cell DNA polymerases. The formation of such episomal concatamers protects the AAV vector during the life of the host cell, thereby allowing for prolonged expression of the transgene in vivo. Suitably, ITR elements will be the only sequences retained from the native AAV genome in the derivative. A derivative will preferably not include the rep and/or cap genes of the native genome and any other sequences of the native genome. This is preferred for the reasons described above, and also to reduce the possibility of integration of the vector into the host cell genome. Additionally, reducing the size of the AAV genome allows for increased flexibility in incorporating other sequence elements (such as regulatory elements) within the vector in addition to the transgene. The following portions could therefore be removed in a derivative of the invention: one inverted terminal repeat (ITR) sequence, the replication (rep) and capsid (cap) genes. However, derivatives may additionally include one or more rep and/or cap genes or other viral sequences of an AAV genome. Naturally occurring AAV integrates with a high frequency at a specific site on human chromosome 19, and shows a negligible frequency of random integration, such that retention of an integrative capacity in the AAV vector may be tolerated in a therapeutic setting. The invention additionally encompasses the provision of sequences of an AAV genome in a different order and configuration to that of a native AAV genome. The invention also encompasses the replacement of one or more AAV sequences or genes with sequences from another virus or with chimeric genes composed of sequences from more than one virus. Such chimeric genes may be composed of sequences from two or more related viral proteins of different viral species. AAV serotype and capsid proteins The AAV vector particle may be encapsidated by capsid proteins. The serotype may facilitate the transduction of glomerular cells (e.g. podocytes), for example, specific transduction of glomerular cells (e.g. podocytes). The AAV vector particle may be a kidney-specific vector particle. Preferably, the AAV vector particle is a glomerular-specific (e.g. podocyte-specific) vector particle. The AAV vector particle may be encapsidated by a glomerular-specific (e.g. podocyte-specific) capsid. The AAV vector particle may comprise a glomerular-specific (e.g. podocyte-specific) capsid protein. Suitably, the AAV vector particles may be transcapsidated forms wherein an AAV genome or derivative having an ITR of one serotype is packaged in the capsid of a different serotype. The AAV vector particle also includes mosaic forms wherein a mixture of unmodified capsid proteins from two or more different serotypes makes up the viral capsid. The AAV vector particle also includes chemically modified forms bearing ligands adsorbed to the capsid surface. For example, such ligands may include antibodies for targeting a particular cell surface receptor. Where a derivative comprises capsid proteins i.e. VP1, VP2 and/or VP3, the derivative may be a chimeric, shuffled or capsid-modified derivative of one or more naturally occurring AAVs. In particular, the invention encompasses the provision of capsid protein sequences from different serotypes, clades, clones, or isolates of AAV within the same vector (i.e. a pseudotyped vector). The AAV vector may be in the form of a pseudotyped AAV vector particle. Chimeric, shuffled or capsid-modified derivatives will be typically selected to provide one or more desired functionalities for the AAV vector. Thus, these derivatives may display increased efficiency of gene delivery, decreased immunogenicity (humoral or cellular), an altered tropism range and/or improved targeting of podocytes compared to an AAV vector comprising a naturally occurring AAV genome. Increased efficiency of gene delivery may be effected by improved receptor or co-receptor binding at the cell surface, improved internalisation, improved trafficking within the cell and into the nucleus, improved uncoating of the viral particle and improved conversion of a single-stranded genome to double-stranded form. Increased efficiency may also relate to an altered tropism range or targeting of podocytes, such that the vector dose is not diluted by administration to tissues where it is not needed. Chimeric capsid proteins include those generated by recombination between two or more capsid coding sequences of naturally occurring AAV serotypes. This may be performed for example by a marker rescue approach in which non-infectious capsid sequences of one serotype are co-transfected with capsid sequences of a different serotype, and directed selection is used to select for capsid sequences having desired properties. The capsid sequences of the different serotypes can be altered by homologous recombination within the cell to produce novel chimeric capsid proteins. Chimeric capsid proteins also include those generated by engineering of capsid protein sequences to transfer specific capsid protein domains, surface loops or specific amino acid residues between two or more capsid proteins, for example between two or more capsid proteins of different serotypes. Shuffled or chimeric capsid proteins may also be generated by DNA shuffling or by error-prone PCR. Hybrid AAV capsid genes can be created by randomly fragmenting the sequences of related AAV genes e.g. those encoding capsid proteins of multiple different serotypes and then subsequently reassembling the fragments in a self-priming polymerase reaction, which may also cause crossovers in regions of sequence homology. A library of hybrid AAV genes created in this way by shuffling the capsid genes of several serotypes can be screened to identify viral clones having a desired functionality. Similarly, error prone PCR may be used to randomly mutate AAV capsid genes to create a diverse library of variants which may then be selected for a desired property. The sequences of the capsid genes may also be genetically modified to introduce specific deletions, substitutions or insertions with respect to the native wild-type sequence. In particular, capsid genes may be modified by the insertion of a sequence of an unrelated protein or peptide within an open reading frame of a capsid coding sequence, or at the N- and/or C-terminus of a capsid coding sequence. The unrelated protein or peptide may advantageously be one which acts as a ligand for a particular cell type, thereby conferring improved binding to a target cell or improving the specificity of targeting of the vector to a particular cell population. The unrelated protein may also be one which assists purification of the viral particle as part of the production process, i.e. an epitope or affinity tag. The site of insertion will typically be selected so as not to interfere with other functions of the viral particle e.g. internalisation, trafficking of the viral particle. The capsid protein may be an artificial or mutant capsid protein. The term “artificial capsid” as used herein means that the capsid particle comprises an amino acid sequence which does not occur in nature or which comprises an amino acid sequence which has been engineered (e.g. modified) from a naturally occurring capsid amino acid sequence. In other words the artificial capsid protein comprises a mutation or a variation in the amino acid sequence compared to the sequence of the parent capsid from which it is derived where the artificial capsid amino acid sequence and the parent capsid amino acid sequences are aligned. The capsid protein may comprise a mutation or modification relative to the wild type capsid protein which improves the ability to transduce podocytes relative to an unmodified or wild type viral particle. Improved ability to transduce podocytes may be measured for example by measuring the expression of a transgene, e.g. GFP, carried by the AAV vector particle, wherein expression of the transgene in podocytes correlates with the ability of the AAV vector particle to transduce podocytes. The AAV vector particle may be an AAV3B, LK03, AAV9, ShH10, AAV-DJ, AAV2, AAV6.2, AAV5, or AAV8 vector particle. In some embodiments, the AAV vector particle is an AAV3B vector particle or an LK03 vector particle. In one embodiment, the AAV vector particle is an LK03 vector particle. In other embodiments, the AAV vector particle is a ShH10, AAV-DJ, AAV2, AAV6.2, or AAV5 vector particle. The AAV vector particle may comprise an AAV3B, LK03, AAV9, ShH10, AAV-DJ, AAV2, AAV6.2, AAV5, or AAV8 capsid protein. In some embodiments, the AAV vector particle comprises an AAV3B capsid protein or an LK03 capsid protein. In one embodiment, the AAV vector particle comprises an LK03 capsid protein. In other embodiments, the AAV vector particle comprises a ShH10, AAV-DJ, AAV2, AAV6.2, or AAV5 capsid protein. The AAV vector particle may comprise AAV3B, LK03, AAV9, ShH10, AAV-DJ, AAV2, AAV6.2, AAV5, or AAV8 capsid proteins VP1, VP2 and VP3. In some embodiments, the AAV vector particle comprises AAV3B or LK03 capsid proteins VP1, VP2 and VP3. In one embodiment, the AAV vector particle comprises LK03 capsid proteins VP1, VP2 and VP3. In other embodiments, the AAV vector particle comprises ShH10, AAV-DJ, AAV2, AAV6.2, or AAV5 capsid proteins VP1, VP2 and VP3. The AAV vector particle may comprise one or more AAV2 ITR sequences and AAV3B capsid proteins, LK03 capsid proteins, AAV9 capsid proteins, ShH10 capsid proteins, AAV-DJ capsid proteins, AAV2 capsid proteins, AAV6.2 capsid proteins, AAV5 capsid proteins, or AAV8 capsid proteins. In some embodiments, the AAV vector particle comprises one or more AAV2 ITR sequences and AAV3B or LK03 capsid proteins. In other embodiments, the AAV vector particle comprises one or more AAV2 ITR sequences and ShH10, AAV-DJ, AAV2, AAV6.2, or AAV5 capsid proteins. The AAV vector particle may have an AAV2 genome and AAV3B capsid proteins (AAV2/3B), an AAV2 genome and LK03 capsid proteins, an AAV2 genome and AAV9 capsid proteins (AAV2/9), an AAV2 genome and ShH10 capsid proteins, an AAV2 genome and AAV-DJ capsid proteins, an AAV2 genome and AAV2 capsid proteins, an AAV2 genome and AAV6.2 capsid proteins, an AAV2 genome and AAV5 capsid proteins, or an AAV2 genome and AAV8 capsid proteins (AAV2/8). The nomenclature AAVX/Y may denote a pseudotyped AAV, for example where the ITR sequences are from AAVX and flank a cassette harbouring a payload which is encapsidated into serotype AAVY (i.e. with AAVY capsid proteins). AAV3B serotype The AAV vector particle may comprise an AAV3B capsid protein. Suitably, the AAV vector particle may be encapsidated by AAV3B capsid proteins. Two distinct AAV3 isolates (AAV3A and AAV3B) have been cloned. In comparison with vectors based on other AAV serotypes, it is thought that AAV3 vectors inefficiently transduce most cell types. However, AAV3B may efficiently transduce podocytes. AA3B has been described in Rutledge, E.A., et al., 1998. Journal of virology, 72(1), pp.309-319. The AAV vector particle may comprise an AAV3B VP1 capsid protein, an AAV3B VP2 capsid protein, and/or an AAV3B VP3 capsid protein. Suitably, the AAV vector particle may be encapsidated by AAV3B VP1 capsid proteins, AAV3B VP2 capsid proteins, and/or AAV3B VP3 capsid proteins. Suitably, the AAV vector particle may be encapsidated by AAV3B VP1, VP2, and VP3 capsid proteins. Suitably, the AAV3B VP1 capsid protein may comprise or consist of the amino acid sequence shown as SEQ ID NO: 26, or a variant which is at least 90% identical to SEQ ID NO: 26. Exemplary AAV3B VP1 capsid protein (SEQ ID NO: 26): MAADGYLPDWLEDNLSEGIREWWALKPGVPQPKANQQHQDNRRGLVLPGYKYLGPGNGLDKGEPVNEA DAAALEHDKAYDQQLKAGDNPYLKYNHADAEFQERLQEDTSFGGNLGRAVFQAKKRILEPLGLVEEAA KTAPGKKRPVDQSPQEPDSSSGVGKSGKQPARKRLNFGQTGDSESVPDPQPLGEPPAAPTSLGSNTMA SGGGAPMADNNEGADGVGNSSGNWHCDSQWLGDRVITTSTRTWALPTYNNHLYKQISSQSGASNDNHY FGYSTPWGYFDFNRFHCHFSPRDWQRLINNNWGFRPKKLSFKLFNIQVKEVTQNDGTTTIANNLTSTV QVFTDSEYQLPYVLGSAHQGCLPPFPADVFMVPQYGYLTLNNGSQAVGRSSFYCLEYFPSQMLRTGNN FQFSYTFEDVPFHSSYAHSQSLDRLMNPLIDQYLYYLNRTQGTTSGTTNQSRLLFSQAGPQSMSLQAR NWLPGPCYRQQRLSKTANDNNNSNFPWTAASKYHLNGRDSLVNPGPAMASHKDDEEKFFPMHGNLIFG KEGTTASNAELDNVMITDEEEIRTTNPVATEQYGTVANNLQSSNTAPTTRTVNDQGALPGMVWQDRDV YLQGPIWAKIPHTDGHFHPSPLMGGFGLKHPPPQIMIKNTPVPANPPTTFSPAKFASFITQYSTGQVS VEIEWELQKENSKRWNPEIQYTSNYNKSVNVDFTVDTNGVYSEPRPIGTRYLTRN Suitably, the variant may be at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 26. Suitably, the AAV3B VP2 and VP3 capsid proteins may be N-terminal truncations of SEQ ID NO: 26, or N-terminal truncations of a variant which is at least 90% identical, at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 26. LK03 serotype The AAV vector particle may comprise an LK03 capsid protein. Suitably, the AAV vector particle may be encapsidated by LK03 capsid proteins. The AAV-LK03 cap sequence consists of fragments from seven different wild-type serotypes (AAV1, 2, 3B, 4, 6, 8, 9) and is described in Lisowski, L., et al., 2014. Nature, 506(7488), pp.382-386. The present inventors have demonstrated that AAV-LK03 vectors can achieve high transduction of close to 100% in human podocytes in vitro. The AAV vector particle may comprise an LK03 VP1 capsid protein, an LK03 VP2 capsid protein, and/or an LK03 VP3 capsid protein. Suitably, the AAV vector particle may be encapsidated by LK03 VP1 capsid proteins, LK03 VP2 capsid proteins, and/or LK03 VP3 capsid proteins. Suitably, the AAV vector particle may be encapsidated by LK03 VP1, VP2, and VP3 capsid proteins. Suitably, the LK03 VP1 capsid protein may comprise or consist of the amino acid sequence shown as SEQ ID NO: 27, or a variant which is at least 90% identical to SEQ ID NO: 27. Exemplary LK03 VP1 capsid protein (SEQ ID NO: 27): MAADGYLPDWLEDNLSEGIREWWALQPGAPKPKANQQHQDNARGLVLPGYKYLGPGNGLDKGEPVNAA DAAALEHDKAYDQQLKAGDNPYLKYNHADAEFQERLKEDTSFGGNLGRAVFQAKKRLLEPLGLVEEAA KTAPGKKRPVDQSPQEPDSSSGVGKSGKQPARKRLNFGQTGDSESVPDPQPLGEPPAAPTSLGSNTMA SGGGAPMADNNEGADGVGNSSGNWHCDSQWLGDRVITTSTRTWALPTYNNHLYKQISSQSGASNDNHY FGYSTPWGYFDFNRFHCHFSPRDWQRLINNNWGFRPKKLSFKLFNIQVKEVTQNDGTTTIANNLTSTV QVFTDSEYQLPYVLGSAHQGCLPPFPADVFMVPQYGYLTLNNGSQAVGRSSFYCLEYFPSQMLRTGNN FQFSYTFEDVPFHSSYAHSQSLDRLMNPLIDQYLYYLNRTQGTTSGTTNQSRLLFSQAGPQSMSLQAR NWLPGPCYRQQRLSKTANDNNNSNFPWTAASKYHLNGRDSLVNPGPAMASHKDDEEKFFPMHGNLIFG KEGTTASNAELDNVMITDEEEIRTTNPVATEQYGTVANNLQSSNTAPTTRTVNDQGALPGMVWQDRDV YLQGPIWAKIPHTDGHFHPSPLMGGFGLKHPPPQIMIKNTPVPANPPTTFSPAKFASFITQYSTGQVS VEIEWELQKENSKRWNPEIQYTSNYNKSVNVDFTVDTNGVYSEPRPIGTRYLTRPL Suitably, the variant may be at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 27. Suitably, the LK03 VP2 and VP3 capsid proteins may be N-terminal truncations of SEQ ID NO: 27, or N-terminal truncations of a variant which is at least 90% identical, at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 27. AAV9 serotype The AAV vector particle may comprise an AAV9 capsid protein. Suitably, the AAV vector particle may be encapsidated by AAV9 capsid proteins. The present inventors have demonstrated that AAV9 vectors can achieve high transduction in human podocytes in vivo. The AAV vector particle may comprise an AAV9 VP1 capsid protein, an AAV9 VP2 capsid protein, and/or an AAV9 VP3 capsid protein. Suitably, the AAV vector particle may be encapsidated by AAV9 VP1 capsid proteins, AAV9 VP2 capsid proteins, and/or AAV9 VP3 capsid proteins. Suitably, the AAV vector particle may be encapsidated by AAV9 VP1, VP2, and VP3 capsid proteins. Suitably, the AAV9 VP1 capsid protein may comprise or consist of the amino acid sequence shown as SEQ ID NO: 28, or a variant which is at least 90% identical to SEQ ID NO: 28. Exemplary AAV9 VP1 capsid protein (SEQ ID NO: 28): MAADGYLPDWLEDNLSEGIREWWALKPGAPQPKANQQHQDNARGLVLPGYKYLGPGNGLDKGEPVNAA DAAALEHDKAYDQQLKAGDNPYLKYNHADAEFQERLKEDTSFGGNLGRAVFQAKKRLLEPLGLVEEAA KTAPGKKRPVEQSPQEPDSSAGIGKSGAQPAKKRLNFGQTGDTESVPDPQPIGEPPAAPSGVGSLTMA SGGGAPVADNNEGADGVGSSSGNWHCDSQWLGDRVITTSTRTWALPTYNNHLYKQISNSTSGGSSNDN AYFGYSTPWGYFDFNRFHCHFSPRDWQRLINNNWGFRPKRLNFKLFNIQVKEVTDNNGVKTIANNLTS TVQVFTDSDYQLPYVLGSAHEGCLPPFPADVFMIPQYGYLTLNDGSQAVGRSSFYCLEYFPSQMLRTG NNFQFSYEFENVPFHSSYAHSQSLDRLMNPLIDQYLYYLSKTINGSGQNQQTLKFSVAGPSNMAVQGR NYIPGPSYRQQRVSTTVTQNNNSEFAWPGASSWALNGRNSLMNPGPAMASHKEGEDRFFPLSGSLIFG KQGTGRDNVDADKVMITNEEEIKTTNPVATESYGQVATNHQSAQAQAQTGWVQNQGILPGMVWQDRDV YLQGPIWAKIPHTDGNFHPSPLMGGFGMKHPPPQILIKNTPVPADPPTAFNKDKLNSFITQYSTGQVS VEIEWELQKENSKRWNPEIQYTSNYYKSNNVEFAVNTEGVYSEPRPIGTRYLTRNL Suitably, the variant may be at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 28. Suitably, the AAV9 VP2 and VP3 capsid proteins may be N-terminal truncations of SEQ ID NO: 28, or N-terminal truncations of a variant which is at least 90% identical, at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 28. ShH10 serotype The AAV vector particle may comprise a ShH10 capsid protein. Suitably, the AAV vector particle may be encapsidated by ShH10 capsid proteins. The ShH10 variant is derived from AAV6 and has increased specificity and efficiency for Müller cells (see e.g. Klimczak, R.R., et al., 2009. PloS one, 4(10), p.e7467). The AAV vector particle may comprise a ShH10 VP1 capsid protein, a ShH10 VP2 capsid protein, and/or a ShH10 VP3 capsid protein. Suitably, the AAV vector particle may be encapsidated by ShH10 VP1 capsid proteins, ShH10 VP2 capsid proteins, and/or ShH10 VP3 capsid proteins. Suitably, the AAV vector particle may be encapsidated by ShH10 VP1, VP2, and VP3 capsid proteins. Suitably, the ShH10 VP1 capsid protein may comprise or consist of the amino acid sequence shown as SEQ ID NO: 29, or a variant which is at least 90% identical to SEQ ID NO: 29. Exemplary ShH10 VP1 capsid protein (SEQ ID NO: 29): MAADGYLPDWLEDNLSEGIREWWDLKPGAPKPKANQQKQDDGRGLVLPGYKYLGPFNGLDKGEPVNAA DAAALEHDKAYDQQLKAGDNPYLRYNHADAEFQERLQEDTSFGGNLGRAVFQAKKRVLEPFGLVEEGA KTAPGKKRPVEQSPQEPDSSSGIGKTGQQPAKKRLNFGQTGDSESVPDPQPLGEPPATPAAVGPTTMA SGGGAPMADNNEGADGVGNASGNWHCDSTWLGDRVITTSTRTWALPTYNNHLYKQISSASTGASNDNH YFGYSTPWGYFDFNRFHCHFSPRDWQRLINNNWGFRPKRLNFKLFNVQVKEVTTNDGVTTIANNLTST VQVFSDSEYQLPYVLGSAHQGCLPPFPADVFMIPQYGYLTLNNGSQAVGRSSFYCLEYFPSQMLRTGN NFTFSYTFEDVPFHSSYAHSQSLDRLMNPLIDQYLYYLNRTQDQSGSAQNKDLLFSRGSPAGMSVQPK NWLPGPCYRQQRVSKTKTDNNNSNFTWTGASKYNLNGRESIINPGTAMASHKDDKNKFFPMSGVMIFG KESAGASNTALDNVMITDEEEIKATNPVATERFGTVAVNLQSSSTDPATGDVHVMGALPGMVWQDRDV YLQGPIWAKIPHTDGHFHPSPLMGGFGLKNPPPQILIKNTPVPANPPAEFSATKFASFITQYSTGQVS VEIEWELQKENSKRWNPEVQYTSNYAKSANVDFTVDNNGLYTEPRPIGTRYLTRPL Suitably, the variant may be at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 29. Suitably, the ShH10 VP2 and VP3 capsid proteins may be N-terminal truncations of SEQ ID NO: 29, or N-terminal truncations of a variant which is at least 90% identical, at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 29. AAV-DJ serotype The AAV vector particle may comprise an AAV-DJ capsid protein. Suitably, the AAV vector particle may be encapsidated by AAV-DJ capsid proteins. AAV-DJ is a hybrid vector created from DNA shuffling of eight AAV serotypes, which mediates efficient gene expression both in vitro and in vivo (see e.g. Mao, Y., et al., 2016. BMC biotechnology, 16, pp.1-8). The AAV vector particle may comprise an AAV-DJ VP1 capsid protein, an AAV-DJ VP2 capsid protein, and/or an AAV-DJ VP3 capsid protein. Suitably, the AAV vector particle may be encapsidated by AAV-DJ VP1 capsid proteins, AAV-DJ VP2 capsid proteins, and/or AAV-DJ VP3 capsid proteins. Suitably, the AAV vector particle may be encapsidated by AAV-DJ VP1, VP2, and VP3 capsid proteins. Suitably, the AAV-DJ VP1 capsid protein may comprise or consist of the amino acid sequence shown as SEQ ID NO: 30, or a variant which is at least 90% identical to SEQ ID NO: 30. Exemplary AAV-DJ VP1 capsid protein (SEQ ID NO: 30): MAADGYLPDWLEDTLSEGIRQWWKLKPGPPPPKPAERHKDDSRGLVLPGYKYLGPFNGLDKGEPVNEA DAAALEHDKAYDRQLDSGDNPYLKYNHADAEFQERLKEDTSFGGNLGRAVFQAKKRLLEPLGLVEEAA KTAPGKKRPVEHSPVEPDSSSGTGKAGQQPARKRLNFGQTGDADSVPDPQPIGEPPAAPSGVGSLTMA AGGGAPMADNNEGADGVGNSSGNWHCDSTWMGDRVITTSTRTWALPTYNNHLYKQISNSTSGGSSNDN AYFGYSTPWGYFDFNRFHCHFSPRDWQRLINNNWGFRPKRLSFKLFNIQVKEVTQNEGTKTIANNLTS TIQVFTDSEYQLPYVLGSAHQGCLPPFPADVFMIPQYGYLTLNNGSQAVGRSSFYCLEYFPSQMLKTG NNFQFTYTFEDVPFHSSYAHSQSLDRLMNPLIDQYLYYLSRTQTTGGTTNTQTLGFSQGGPNTMANQA KNWLPGPCYRQQRVSKTSADNNNSEYSWTGATKYHLNGRDSLVNPGPAMASHKDDEEKFFPQSGVLIF GKQGSEKTNVDIEKVMITDEEEIRTTNPVATEQYGSVSTNLQRGNRQAATADVNTQGVLPGMVWQDRD VYLQGPIWAKIPHTDGHFHPSPLMGGFGLKHPPPQILIKNTPVPADPPTTFNQSKLNSFITQYSTGQV SVEIEWELQKENSKRWNPEIQYTSNYYKSTSVDFAVNTEGVYSEPRPIGTRYLTRNL Suitably, the variant may be at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 30. Suitably, the AAV-DJ VP2 and VP3 capsid proteins may be N-terminal truncations of SEQ ID NO: 30, or N-terminal truncations of a variant which is at least 90% identical, at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 30. AAV2 serotype The AAV vector particle may comprise an AAV2 capsid protein. Suitably, the AAV vector particle may be encapsidated by AAV2 capsid proteins. The AAV vector particle may comprise an AAV2 VP1 capsid protein, an AAV2 VP2 capsid protein, and/or an AAV2 VP3 capsid protein. Suitably, the AAV vector particle may be encapsidated by AAV2 VP1 capsid proteins, AAV2 VP2 capsid proteins, and/or AAV2 VP3 capsid proteins. Suitably, the AAV vector particle may be encapsidated by AAV2 VP1, VP2, and VP3 capsid proteins. Suitably, the AAV2 VP1 capsid protein may comprise or consist of the amino acid sequence shown as SEQ ID NO: 31, or a variant which is at least 90% identical to SEQ ID NO: 31. Exemplary AAV2 VP1 capsid protein (SEQ ID NO: 31): MAADGYLPDWLEDTLSEGIRQWWKLKPGPPPPKPAERHKDDSRGLVLPGYKYLGPFNGLDKGEPVNEA DAAALEHDKAYDRQLDSGDNPYLKYNHADAEFQERLKEDTSFGGNLGRAVFQAKKRVLEPLGLVEEPV KTAPGKKRPVEHSPVEPDSSSGTGKAGQQPARKRLNFGQTGDADSVPDPQPLGQPPAAPSGLGTNTMA TGSGAPMADNNEGADGVGNSSGNWHCDSTWMGDRVITTSTRTWALPTYNNHLYKQISSQSGASNDNHY FGYSTPWGYFDFNRFHCHFSPRDWQRLINNNWGFRPKRLNFKLFNIQVKEVTQNDGTTTIANNLTSTV QVFTDSEYQLPYVLGSAHQGCLPPFPADVFMVPQYGYLTLNNGSQAVGRSSFYCLEYFPSQMLRTGNN FTFSYTFEDVPFHSSYAHSQSLDRLMNPLIDQYLYYLSRTNTPSGTTTQSRLQFSQAGASDIRDQSRN WLPGPCYRQQRVSKTSADNNNSEYSWTGATKYHLNGRDSLVNPGPAMASHKDDEEKFFPQSGVLIFGK QGSEKTNVDIEKVMITDEEEIRTTNPVATEQYGSVSTNLQRGNRQAATADVNTQGVLPGMVWQDRDVY LQGPIWAKIPHTDGHFHPSPLMGGFGLKHPPPQILIKNTPVPANPSTTFSAAKFASFITQYSTGQVSV EIEWELQKENSKRWNPEIQYTSNYNKSVNRGLTVDTNGVYSEPRPIGTRYLTRNL Suitably, the variant may be at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 31. Suitably, the AAV2 VP2 and VP3 capsid proteins may be N-terminal truncations of SEQ ID NO: 31, or N-terminal truncations of a variant which is at least 90% identical, at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 31. AAV6.2 serotype The AAV vector particle may comprise an AAV6.2 capsid protein. Suitably, the AAV vector particle may be encapsidated by AAV6.2 capsid proteins. The AAV6.2 vector mutant was created by mutating the phenyalanine (F) residue at position 129 in AAV6 to leucine (L) (see e.g. Limberis, M.P., et al., 2009. Molecular Therapy, 17(2), pp.294-301). The AAV vector particle may comprise an AAV6.2 VP1 capsid protein, an AAV6.2 VP2 capsid protein, and/or an AAV6.2 VP3 capsid protein. Suitably, the AAV vector particle may be encapsidated by AAV6.2 VP1 capsid proteins, AAV6.2 VP2 capsid proteins, and/or AAV6.2 VP3 capsid proteins. Suitably, the AAV vector particle may be encapsidated by AAV6.2 VP1, VP2, and VP3 capsid proteins. Suitably, the AAV6.2 VP1 capsid protein may comprise or consist of the amino acid sequence shown as SEQ ID NO: 32, or a variant which is at least 90% identical to SEQ ID NO: 32. Exemplary AAV6.2 VP1 capsid protein (SEQ ID NO: 32): MAADGYLPDWLEDNLSEGIREWWDLKPGAPKPKANQQKQDDGRGLVLPGYKYLGPFNGLDKGEPVNAA DAAALEHDKAYDQQLKAGDNPYLRYNHADAEFQERLQEDTSFGGNLGRAVFQAKKRVLEPLGLVEEGA KTAPGKKRPVEQSPQEPDSSSGIGKTGQQPAKKRLNFGQTGDSESVPDPQPLGEPPATPAAVGPTTMA SGGGAPMADNNEGADGVGNASGNWHCDSTWLGDRVITTSTRTWALPTYNNHLYKQISSASTGASNDNH YFGYSTPWGYFDFNRFHCHFSPRDWQRLINNNWGFRPKRLNFKLFNIQVKEVTTNDGVTTIANNLTST VQVFSDSEYQLPYVLGSAHQGCLPPFPADVFMIPQYGYLTLNNGSQAVGRSSFYCLEYFPSQMLRTGN NFTFSYTFEDVPFHSSYAHSQSLDRLMNPLIDQYLYYLNRTQNQSGSAQNKDLLFSRGSPAGMSVQPK NWLPGPCYRQQRVSKTKTDNNNSNFTWTGASKYNLNGRESIINPGTAMASHKDDKDKFFPMSGVMIFG KESAGASNTALDNVMITDEEEIKATNPVATERFGTVAVNLQSSSTDPATGDVHVMGALPGMVWQDRDV YLQGPIWAKIPHTDGHFHPSPLMGGFGLKHPPPQILIKNTPVPANPPAEFSATKFASFITQYSTGQVS VEIEWELQKENSKRWNPEVQYTSNYAKSANVDFTVDNNGLYTEPRPIGTRYLTRPL Suitably, the variant may be at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 32. Suitably, the AAV6.2 VP2 and VP3 capsid proteins may be N-terminal truncations of SEQ ID NO: 32, or N-terminal truncations of a variant which is at least 90% identical, at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 32. AAV5 serotype The AAV vector particle may comprise an AAV5 capsid protein. Suitably, the AAV vector particle may be encapsidated by AAV5 capsid proteins. The AAV vector particle may comprise an AAV5 VP1 capsid protein, an AAV5 VP2 capsid protein, and/or an AAV5 VP3 capsid protein. Suitably, the AAV vector particle may be encapsidated by AAV5 VP1 capsid proteins, AAV5 VP2 capsid proteins, and/or AAV5 VP3 capsid proteins. Suitably, the AAV vector particle may be encapsidated by AAV5 VP1, VP2, and VP3 capsid proteins. Suitably, the AAV5 VP1 capsid protein may comprise or consist of the amino acid sequence shown as SEQ ID NO: 33, or a variant which is at least 90% identical to SEQ ID NO: 33. Exemplary AAV5 VP1 capsid protein (SEQ ID NO: 33): MSFVDHPPDWLEEVGEGLREFLGLEAGPPKPKPNQQHQDQARGLVLPGYNYLGPGNGLDRGEPVNRAD EVAREHDISYNEQLEAGDNPYLKYNHADAEFQEKLADDTSFGGNLGKAVFQAKKRVLEPFGLVEEGAK TAPTGKRIDDHFPKRKKARTEEDSKPSTSSDAEAGPSGSQQLQIPAQPASSLGADTMSAGGGGPLGDN NQGADGVGNASGDWHCDSTWMGDRVVTKSTRTWVLPSYNNHQYREIKSGSVDGSNANAYFGYSTPWGY FDFNRFHSHWSPRDWQRLINNYWGFRPRSLRVKIFNIQVKEVTVQDSTTTIANNLTSTVQVFTDDDYQ LPYVVGNGTEGCLPAFPPQVFTLPQYGYATLNRDNTENPTERSSFFCLEYFPSKMLRTGNNFEFTYNF EEVPFHSSFAPSQNLFKLANPLVDQYLYRFVSTNNTGGVQFNKNLAGRYANTYKNWFPGPMGRTQGWN LGSGVNRASVSAFATTNRMELEGASYQVPPQPNGMTNNLQGSNTYALENTMIFNSQPANPGTTATYLE GNMLITSESETQPVNRVAYNVGGQMATNNQSSTTAPATGTYNLQEIVPGSVWMERDVYLQGPIWAKIP ETGAHFHPSPAMGGFGLKHPPPMMLIKNTPVPGNITSFSDVPVSSFITQYSTGQVTVEMEWELKKENS KRWNPEIQYTNNYNDPQFVDFAPDSTGEYRTTRPIGTRYLTRPL Suitably, the variant may be at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 33. Suitably, the AAV5 VP2 and VP3 capsid proteins may be N-terminal truncations of SEQ ID NO: 33, or N-terminal truncations of a variant which is at least 90% identical, at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to SEQ ID NO: 33. Variants, derivatives, analogues, homologues and fragments In addition to the specific proteins (e.g., encoded by the transgenes) and nucleotides (e.g., promoters) mentioned herein, the invention also encompasses variants, derivatives, homologues and fragments thereof. In the context of the invention, a “variant” of any given sequence is a sequence in which the specific sequence of residues (whether amino acid or nucleic acid residues) has been modified in such a manner that the polypeptide or polynucleotide in question retains at least one of its endogenous functions. A variant sequence can be obtained by addition, deletion, substitution, modification, replacement and/or variation of at least one residue present in the naturally occurring polypeptide or polynucleotide. For example, a variant promoter sequence retains at least some level of the activity and specificity of the promoter sequence from which it is obtained. The term “derivative” as used herein in relation to proteins or polypeptides of the invention includes any substitution of, variation of, modification of, replacement of, deletion of and/or addition of one (or more) amino acid residues from or to the sequence, providing that the resultant protein or polypeptide retains at least one of its endogenous functions. Typically, amino acid substitutions may be made, for example from 1, 2 or 3, to 10 or 20 substitutions, provided that the modified sequence retains the required activity or ability. Amino acid substitutions may include the use of non-naturally occurring analogues. Typically, nucleic acid substitutions may be made, for example from 1, 2 or 3, to 10 or 20 substitutions (e.g.1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, or 20 substitutions), provided that the modified sequence retains the required activity or ability. Proteins used in the invention may also have deletions, insertions or substitutions of amino acid residues which produce a silent change and result in a functionally equivalent protein. Deliberate amino acid substitutions may be made on the basis of similarity in polarity, charge, solubility, hydrophobicity, hydrophilicity and/or the amphipathic nature of the residues as long as the endogenous function is retained. For example, negatively charged amino acids include aspartic acid and glutamic acid; positively charged amino acids include lysine and arginine; and amino acids with uncharged polar head groups having similar hydrophilicity values include asparagine, glutamine, serine, threonine and tyrosine. Conservative substitutions may be made, for example according to the table below. Amino acids in the same block in the second column and preferably in the same line in the third column may be substituted for each other: The term “homologue” as used herein means a variant having a certain homology with the wild type amino acid sequence or the wild type nucleotide sequence. The term “homology” can be equated with “identity”. In the present context, a homologous sequence is taken to include an amino acid sequence which may be at least 50%, 55%, 65%, 75%, 85% or 90% identical, preferably at least 95%, 96% or 97% or 98% or 99% identical to the subject sequence. Typically, the homologues will comprise the same active sites etc. as the subject amino acid sequence. Although homology can also be considered in terms of similarity (i.e. amino acid residues having similar chemical properties/functions), in the context of the present invention it is preferred to express homology in terms of sequence identity. In the present context, a homologous sequence is taken to include a nucleotide sequence which may be at least 50%, 55%, 65%, 75%, 85% or 90% identical, preferably at least 95%, 96% or 97% or 98% or 99% identical to the subject sequence. Although homology can also be considered in terms of similarity, in the context of the present invention it is preferred to express homology in terms of sequence identity. Preferably, reference to a sequence which has a percent identity to any one of the SEQ ID NOs detailed herein refers to a sequence which has the stated percent identity over the entire length of the SEQ ID NO referred to. Homology comparisons can be conducted by eye, or more usually, with the aid of readily available sequence comparison programs. These commercially available computer programs can calculate percent homology or identity between two or more sequences. Percent homology may be calculated over contiguous sequences, i.e. one sequence is aligned with the other sequence and each amino acid or nucleotide in one sequence is directly compared with the corresponding amino acid or nucleotide in the other sequence, one residue at a time. This is called an “ungapped” alignment. Typically, such ungapped alignments are performed only over a relatively short number of residues. Although this is a very simple and consistent method, it fails to take into consideration that, for example, in an otherwise identical pair of sequences, one insertion or deletion in the amino acid or nucleotide sequence may cause the following residues or codons to be put out of alignment, thus potentially resulting in a large reduction in percent homology when a global alignment is performed. Consequently, most sequence comparison methods are designed to produce optimal alignments that take into consideration possible insertions and deletions without penalising unduly the overall homology score. This is achieved by inserting “gaps” in the sequence alignment to try to maximise local homology. However, these more complex methods assign “gap penalties” to each gap that occurs in the alignment so that, for the same number of identical amino acids or nucleotides, a sequence alignment with as few gaps as possible, reflecting higher relatedness between the two compared sequences, will achieve a higher score than one with many gaps. “Affine gap costs” are typically used that charge a relatively high cost for the existence of a gap and a smaller penalty for each subsequent residue in the gap. This is the most commonly used gap scoring system. High gap penalties will of course produce optimised alignments with fewer gaps. Most alignment programs allow the gap penalties to be modified. However, it is preferred to use the default values when using such software for sequence comparisons. For example when using the GCG Wisconsin Bestfit package the default gap penalty for amino acid sequences is -12 for a gap and -4 for each extension. Calculation of maximum percent homology therefore firstly requires the production of an optimal alignment, taking into consideration gap penalties. A suitable computer program for carrying out such an alignment is the GCG Wisconsin Bestfit package (University of Wisconsin, USA; Devereux et al. (1984) Nucleic Acids Research 12: 387). Examples of other software that can perform sequence comparisons include, but are not limited to, the BLAST package (see Ausubel et al. (1999) ibid – Ch.18), FASTA (Atschul et al. (1990) J. Mol. Biol. 403-410), EMBOSS Needle (Madeira, F., et al., 2019. Nucleic acids research, 47(W1), pp.W636-W641) and the GENEWORKS suite of comparison tools. Both BLAST and FASTA are available for offline and online searching (see Ausubel et al. (1999) ibid, pages 7-58 to 7- 60). However, for some applications, it is preferred to use the GCG Bestfit program. Another tool, BLAST 2 Sequences, is also available for comparing protein and nucleotide sequences (FEMS Microbiol. Lett. (1999) 174(2):247-50; FEMS Microbiol. Lett. (1999) 177(1):187-8). Although the final percent homology can be measured in terms of identity, the alignment process itself is typically not based on an all-or-nothing pair comparison. Instead, a scaled similarity score matrix is generally used that assigns scores to each pairwise comparison based on chemical similarity or evolutionary distance. An example of such a matrix commonly used is the BLOSUM62 matrix (the default matrix for the BLAST suite of programs). GCG Wisconsin programs generally use either the public default values or a custom symbol comparison table if supplied (see the user manual for further details). For some applications, it is preferred to use the public default values for the GCG package, or in the case of other software, the default matrix, such as BLOSUM62. Once the software has produced an optimal alignment, it is possible to calculate percent homology, preferably percent sequence identity. The software typically does this as part of the sequence comparison and generates a numerical result. The percent sequence identity may be calculated as the number of identical residues as a percentage of the total residues in the SEQ ID NO referred to. “Fragments” are also variants and the term typically refers to a selected region of the polypeptide or polynucleotide that is of interest either functionally or, for example, in an assay. “Fragment” thus refers to an amino acid or nucleic acid sequence that is a portion of a full- length polypeptide or polynucleotide. Such variants, derivatives, homologues and fragments may be prepared using standard recombinant DNA techniques such as site-directed mutagenesis. Where insertions are to be made, synthetic DNA encoding the insertion together with 5’ and 3’ flanking regions corresponding to the naturally-occurring sequence either side of the insertion site may be made. The flanking regions will contain convenient restriction sites corresponding to sites in the naturally-occurring sequence so that the sequence may be cut with the appropriate enzyme(s) and the synthetic DNA ligated into the cut. The DNA is then expressed in accordance with the invention to make the encoded protein. These methods are only illustrative of the numerous standard techniques known in the art for manipulation of DNA sequences and other known techniques may also be used. Cells In one aspect, the present invention provides a cell comprising the promoter, polynucleotide, or vector described herein. The cell may be an isolated cell. The cell may be a human cell, suitably an isolated human cell. The cell may be any cell type known in the prior art. Suitably, the cell may be a kidney cell or glomerular cell, for example a podocyte. Suitably, the cell may be an immortalized kidney cell or glomerular cell, for example an immortalized podocyte. Suitable podocyte cell lines will be well known to those of skill in the art, for example CIHP-1. Methods to generate immortalized podocytes will be well known to those of skill in the art. Suitable methods are described in Ni, L., et al., 2012. Nephrology, 17(6), pp.525-531. Suitably, the cell may be a producer cell. The term “producer cell” includes a cell that produces viral particles, after transient transfection, stable transfection or vector transduction of all the elements necessary to produce the viral particles or any cell engineered to stably comprise the elements necessary to produce the viral particles. Suitable producer cells will be well known to those of skill in the art. Suitable producer cell lines include HEK 293 (e.g. HEK 293T), HeLa, and A549 cell lines. Suitably, the cell may be a packaging cell. The term “packaging cell” includes a cell which contains some or all of the elements necessary for packaging an infectious recombinant virus. The packaging cell may lack a recombinant viral vector genome. Typically, such packaging cells contain one or more vectors which are capable of expressing viral structural proteins. Cells comprising only some of the elements required for the production of enveloped viral particles are useful as intermediate reagents in the generation of viral particle producer cell lines, through subsequent steps of transient transfection, transduction or stable integration of each additional required element. These intermediate reagents are encompassed by the term “packaging cell”. Suitable packaging cells will be well known to those of skill in the art. Pharmaceutical composition In one aspect, the present invention provides a pharmaceutical composition comprising the polynucleotide, vector, or cell defined herein. Preferably, the vector is an AAV vector particle. A pharmaceutical composition is a composition that comprises or consists of a therapeutically effective amount of a pharmaceutically active agent i.e. the AAV vector. It preferably includes a pharmaceutically acceptable carrier, diluent or excipient (including combinations thereof). By “pharmaceutically acceptable” is included that the formulation is sterile and pyrogen free. The carrier, diluent, and/or excipient must be “acceptable” in the sense of being compatible with the vector and not deleterious to the recipients thereof. Typically, the carriers, diluents, and excipients will be saline or infusion media which will be sterile and pyrogen free; however, other acceptable carriers, diluents, and excipients may be used. Acceptable carriers, diluents, and excipients for therapeutic use are well known in the pharmaceutical art. The choice of pharmaceutical carrier, excipient or diluent can be selected with regard to the intended route of administration and standard pharmaceutical practice. The pharmaceutical compositions may comprise as - or in addition to - the carrier, excipient or diluent any suitable binder(s), lubricant(s), suspending agent(s), coating agent(s) or solubilising agent(s). Examples of pharmaceutically acceptable carriers include, for example, water, salt solutions, alcohol, silicone, waxes, petroleum jelly, vegetable oils, polyethylene glycols, propylene glycol, liposomes, sugars, gelatin, lactose, amylose, magnesium stearate, talc, surfactants, silicic acid, viscous paraffin, perfume oil, fatty acid monoglycerides and diglycerides, petroethral fatty acid esters, hydroxymethyl-cellulose, polyvinylpyrrolidone, and the like. The vector, cell, or pharmaceutical composition according to the present invention may be administered in a manner appropriate for treating and/or preventing the diseases described herein. The quantity and frequency of administration will be determined by such factors as the condition of the subject, and the type and severity of the subject's disease, although appropriate dosages may be determined by clinical trials. The pharmaceutical composition may be formulated accordingly. The vector, cell or pharmaceutical composition according to the present invention may be administered parenterally, for example, intravenously, or by infusion techniques. The vector, cell or pharmaceutical composition may be administered in the form of a sterile aqueous solution which may contain other substances, for example, enough salts or glucose to make the solution isotonic with blood. The aqueous solution may be suitably buffered (preferably to a pH of from 3 to 9). The pharmaceutical composition may be formulated accordingly. The preparation of suitable parenteral formulations under sterile conditions is readily accomplished by standard pharmaceutical techniques well-known to those skilled in the art. The vector, cell or pharmaceutical composition according to the present invention may be administered systemically, for example by intravenous injection. The vector, cell or pharmaceutical composition according to the present invention may be administered locally, for example by targeting administration to the kidney. Suitably, the vector, cell or pharmaceutical composition may be administered by injection into the renal artery or by ureteral or subcapsular injection. The pharmaceutical compositions may comprise vectors or cells of the invention in infusion media, for example sterile isotonic solution. The pharmaceutical composition may be enclosed in ampoules, disposable syringes or multiple dose vials made of glass or plastic. The vector, cell or pharmaceutical composition may be administered in a single or in multiple doses. Particularly, the vector, cell or pharmaceutical composition may be administered in a single, one-off dose. The pharmaceutical composition may be formulated accordingly. The vector, cell or pharmaceutical composition may be administered at varying doses (e.g. measured in vector genomes (vg) per kg). The physician in any event will determine the actual dosage which will be most suitable for any individual subject and it will vary with the age, weight and response of the particular subject. Typically, however, for the AAV vectors of the invention, doses of 106 to 1014 vg/kg, 107 to 1014 vg/kg, 108 to 1014 vg/kg, 109 to 1014 vg/kg, 1010 to 1014 vg/kg, or 1011 to 1013 vg/kg may be administered. The pharmaceutical composition may further comprise one or more other therapeutic agents. The invention further includes the use of kits comprising the vector, cells and/or pharmaceutical composition of the present invention. Preferably said kits are for use in the methods and used as described herein, e.g., the therapeutic methods as described herein. Preferably, said kits comprise instructions for use of the kit components. Methods for treating and/or preventing disease In one aspect, the present invention provides the vector, cell or pharmaceutical composition described herein for use as a medicament. In one aspect, the present invention provides use of the vector, cell or pharmaceutical composition described herein in the manufacture of a medicament. In one aspect, the present invention provides a method of administering the vector, cell or pharmaceutical composition described herein to a subject in need thereof. Glomerular diseases The vector, cell or pharmaceutical composition described herein may be used to treat glomerular diseases in a subject. Suitably, the subject is a human subject. In one aspect, the present invention provides the vector, cell or pharmaceutical composition described herein for use in preventing or treating a kidney disease, for example a glomerular disease. In a related aspect, the present invention provides use of the vector, cell or pharmaceutical composition described herein for the manufacture of a medicament for preventing or treating a glomerular disease. In a related aspect, the present invention provides a method of preventing or treating a glomerular disease comprising administering the vector, cell or pharmaceutical composition described herein to a subject in need thereof. Glomerular diseases can be classified as either nephrotic or nephritic. Nephrotic syndrome often involves factors that affect the integrity of podocyte–podocyte or podocyte–GBM interactions. By contrast, factors involved in the etiology of nephritic syndrome can vary, but may include circulating platelets and white blood cells, the GBM, resident glomerular endothelial cells, and mesangial cells. (Chiang, C.K. and Inagi, R., 2010. Nature Reviews Nephrology, 6(9), p.539). Suitably, the glomerular disease is a genetic glomerular disease, i.e. a glomerular disease which is inherited. Genetic glomerular diseases include podocyte-associated genetic glomerular diseases, such as nephrotic syndrome, and GBM-associated glomerular diseases, such as Alport Syndrome. Suitably, the glomerular disease is a podocyte-associated genetic glomerular disease. Podocyte-associated genetic glomerular diseases include Congenital nephrotic syndrome of the Finnish type, Congenital nephrotic syndrome type 2, Familial nephrotic syndrome type 3, Frasier syndrome and Denys–Drash syndrome, Schimke immuno-osseous dysplasia, Nephrotic syndrome caused by mutations in CD2AP, Nephrotic syndrome caused by mutations in actinin-4, Nephrotic syndrome caused by mutations in TRPC6, and Epstein and Fechtner syndrome. Suitably, the glomerular disease is nephrotic syndrome. Suitably, the glomerular disease is a GBM-associated genetic glomerular disease. Podocyte-associated genetic glomerular diseases include X-linked Alport syndrome, Autosomal recessive Alport syndrome, Autosomal dominant Alport syndrome, Thin basement membrane diseases, Pierson syndrome, and Nail–patella syndrome. Suitably, the glomerular disease is Alport syndrome (AS). AS is also known as familial nephritis, hereditary nephritis, thin basement membrane disease and thin basement membrane nephropathy. EXAMPLES The invention will now be further described by way of Examples, which are meant to serve to assist one of ordinary skill in the art in carrying out the invention and are not intended in any way to limit the scope of the invention. Example 1 – Transfection of HEK293T cells The following constructs were tested: ^ A construct comprising the full length NPHS2 promoter - NPHS2(FL)-eGFP (see Figure 4A). ^ A construct comprising the 5’ truncated NPHS2 promoter shown in SEQ ID NO: 2 - NPHS2(628bp)-eGFP (see Figure 4B). The sequence of the full length NPHS2 promoter (NPHS2(FL)) used is shown in SEQ ID NO: 1. The sequence of the 5’ truncated NPHS2 promoter (NPHS2(628bp)) used is shown in SEQ ID NO: 2. Materials & Methods: HEK293T cells were seeded at a density of 1e6 cells per well of a 6-well plate and allowed to adhere overnight. Cells were cultured in DMEM supplemented with 10%FBS, in a total volume of 2mls. The following day cells were transfected as follows: 1ug of DNA was added to DMEM to a total volume of 150ul. In a separate tube, 3ul of PEI was added to 147ul DMEM. The PEI/DMEM was then added to the tube containing the DNA/DMEM and the solution was mixed by pipetting up and down ten times. This was incubated for 15 minutes at room temperature and then added dropwise to the well to be transfected. Experiment was performed once with three technical replicates per sample. 48 hours following transfection, cells were analysed by FACS for GFP expression. Briefly, for each sample, media was removed from cells and cells were washed with 1ml of PBS. Following removal of PBS, 500ul of Trypsin was added to cells and incubated at 37 degrees for 5 minutes. Once cells were detached, trypsin was neutralised with 500ul DMEM supplemented with 10% FBS. Cells were centrifuged at 400g for 5 minutes, after which cells were resuspended with 1ml of PBS. Cells were then passed through the cytometer, gating on the whole cell population (SSC-A/FSC-A), followed by singlets (FSC-A/FSC-H) and finally on GFP positive cells (B530/FSC-A). Non-transfected cells were used as the negative control to gate the GFP negative population. As can be seen from Figure 1, both FL and 628bp promoters drive GFP expression in HEK293T cells. Example 2 – Transfection of mouse podocytes The following constructs were tested: NPHS2(FL)-eGFP (see Figure 4A) NPHS2(628bp)-eGFP (see Figure 4B) Materials & Methods: Mouse podocytes were seeded at a density of 5e5 cells per well of a 6-well plate and allowed to adhere overnight. Cells were cultured in RPMI supplemented with 10%FBS and 1% ITS (Insulin, Transferrin & Selenium), in a total volume of 2mls. The following day cells were transfected as follows: 1ug of DNA was added to DMEM to a total volume of 150ul. In a separate tube, 3ul of PEI was added to 147ul DMEM. The PEI/DMEM was then added to the tube containing the DNA/DMEM and the solution was mixed by pipetting up and down ten times. This was incubated for 15 minutes at room temperature and then added dropwise to the well to be transfected. Experiment was performed once with three technical replicates per sample. 48 hours following transfection, cells were analysed by FACS for GFP expression. Briefly, for each sample, media was removed from cells and cells were washed with 1ml of PBS. Following removal of PBS, 500ul of Trypsin was added to cells and incubated at 37 degrees for 5 minutes. Once cells were detached, trypsin was neutralised with 500ul DMEM supplemented with 10% FBS. Cells were centrifuged at 400g for 5 minutes, after which cells were resuspended with 1ml of PBS. Cells were then passed through the cytometer, gating on the whole cell population (SSC-A/FSC-A), followed by singlets (FSC-A/FSC-H) and finally on GFP positive cells (B530/FSC-A). Non-transfected cells were used as the negative control to gate the GFP negative population. As can be seen from Figure 2, both FL and 628bp promoters drive GFP expression in mouse podocytes. Example 3 – Transfection of HEK293T cells The following constructs were tested: NPHS2(FL)-luciferase (see Figure 4C) NPHS2(628bp)-luciferase (see Figure 4D) Materials & Methods: HEK293T cells were seeded at a density of 1e6 cells per well of a 6-well plate and allowed to adhere overnight. Cells were cultured in DMEM supplemented with 10%FBS, in a total volume of 2mls. The following day cells were transfected as follows: 1ug of DNA was added to DMEM to a total volume of 150ul. In a separate tube, 3ul of PEI was added to 147ul DMEM. The PEI/DMEM was then added to the tube containing the DNA/DMEM and the solution was mixed by pipetting up and down ten times. This was incubated for 15 minutes at room temperature and then added dropwise to the well to be transfected. Transfections were performed in duplicate, and three separate experiments were performed (n=3). To assess gene expression a luciferase assay was performed 48 hours following transfection using the Firefly Luciferase Glow Assay kit from Invitrogen (Cat. # 16177). Briefly, for each sample, media was removed from cells and cells were washed with 1ml of PBS. Following removal of PBS, 500ul of Trypsin was added to cells and incubated at 37 degrees for 5 minutes. Once cells were detached, trypsin was neutralised with 500ul DMEM supplemented with 10% FBS. Cells were centrifuged at 400g for 5 minutes, after which cells were resuspended with 1ml of PBS. From this ml of PBS, 100ul was added to a 96-well plate, to which 100ul of 2X lysis buffer (supplied in the kit) was added. Cells were mixed by pipetting and lysis was allowed to occur for 15 minutes on a plate shaker set at 400rpm. Following cell lysis, 20ul of lysis was added to a black 96-well plate for luminescence assay. To this 20ul, 50ul of luciferin detection reagent (supplied with the kit) was added. This was incubated in the dark for 10 minutes, after which luminescence was measured on the plate reader using the luminosity setting. Results are expressed in relative luminescence units (RLU). As can be seen from Figure 3, this experiment demonstrates that both the full length and 628bp NPHS2 promoters drive similar levels of luciferase expression in HEK293T cells. Example 4 – Transfection of AD293 cells Plasmids were constructed comprising a luciferase transgene under the control of the following promoters: Luciferase activity was determined following transfection into AD293 cells. The results are shown in Figure 5. Each of the plasmids had increased luciferase activity compared to the non-transfected cells, demonstrating that each of the 5’ truncated podocin promoters was capable of driving luciferase expression. The dotted line in Figure 5 indicates the mean luciferase activity of a plasmid comprising the control 628bp promoter (PS0471). The PS0576, PS0582, and PS0589 plasmids had significantly higher luciferase activity compared to PS0471 (one-way ANOVA). Materials and methods AD293 Cell Culture AD293 cells were maintained in DMEM supplemented with 10% FBS. Cells were passaged at a ratio of 1:10 once confluency reached approximately 80%. AD293 Transfections For transfections, cells were seeded at a density of 2e5 cells per well of a 6-well plate and allowed to adhere overnight. The following day transfections were set up as follows; a master mix of PEI/DMEM was made by mixing 120ul of PEI with 4.38mls of DMEM. For each plasmid, 2ug of DNA was mixed with DMEM to a final volume of 150ul. To each plasmid/DMEM mix, 150ul PEI mastermix was added and mixed 10 times by pipetting. This was incubated for 20 minutes at room temperature and then the total 300ul was added to a well of a 6-well plate. Media was changed the following morning, and cells harvested for luciferase assay a further 24 hours later. Luciferase assay Luciferase activity was assess using Pierce™ Firefly Luciferase Glow Assay Kit (Thermo Fisher Cat# 16176). Briefly, media was removed from the cells followed by washing the cells once with PBS. 500ul of lysis buffer supplemented with protease/phosphatase inhibitor cocktail (Roche Cat# 11836170001) was added to cells which were then placed on a rotary shaker at 400rpm for 15 minutes to lyse cells.20ul of cell lysis was then used for luciferase assay as per manufacturers guidelines. Samples were run in triplicate for technical replicates. The experiment was performed three times for n=3. The graph provided plotted the average results from technical replicates for three separate experiments. One-way ANOVA was performed for statistical analysis, comparing all samples to the plasmid containing the 628bp NPHS2 promoter. EMBODIMENTS Various preferred features and embodiments of the present invention will now be described with reference to the following numbered paragraphs (paras). 1. An AAV vector comprising a transgene for use in gene therapy, wherein said AAV vector is administered to a subject, wherein the transgene is expressed in podocytes of said subject, and wherein the transgene is operably linked to a 5’-truncated podocin promoter. 2. Use of an AAV vector comprising a transgene in the manufacture of a medicament for gene therapy, wherein said AAV vector is administered to a subject, wherein the transgene is expressed in podocytes of said subject, and wherein the transgene is operably linked to a 5’- truncated podocin promoter. 3. A method of expressing a transgene in podocytes of a subject, the method comprising administering an AAV vector comprising the transgene to said subject, wherein the transgene is expressed in podocytes of said subject, and wherein the transgene is operably linked to a 5’-truncated podocin promoter. 4. The AAV vector for use according to para 1, the use according to para 2, or the method according to para 3, wherein the 5’-truncated podocin promoter is a truncate of a full-length podocin promoter consisting of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 1. 5. The AAV vector for use according to para 1 or 4, the use according to para 2 or 4, or the method according to para 3 or 4, wherein the 5’-truncated podocin promoter has a length of about 1.5 kb or less, about 1.4 kb or less, about 1.3 kb or less, about 1.2 kb or less, about 1.1 kb or less, about 1.0 kb or less, about 0.9 kb or less, about 0.8 kb or less, about 0.7 kb or less, or about 0.6 kb or less. 6. The AAV vector for use according to any of paras 1 or 4-5, the use according to any of paras 2 or 4-5, or the method according to any of paras 3 or 4-5, wherein the 5’-truncated podocin promoter has a length of 628 bp or less. 7. The AAV vector for use according to any of paras 1 or 4-6, the use according to any of paras 2 or 4-6, or the method according to any of paras 3 or 4-6, wherein the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 2. 8. The AAV vector for use according to any of paras 1 or 4-7, the use according to any of paras 2 or 4-7, or the method according to any of paras 3 or 4-7, wherein the AAV vector is administered to the subject in the form of an AAV vector particle. 9. The AAV vector for use according to para 8, the use according to para 8, or the method according to para 8, wherein the AAV vector particle is encapsidated by AAV3B, LK03, or AAV9 capsid proteins. 10. The AAV vector for use according to para 8, the use according to para 8, or the method according to para 8, wherein the AAV vector particle is encapsidated by LK03 capsid proteins. 11. The AAV vector for use according to para 8, the use according to para 8, or the method according to para 8, wherein the AAV vector particle is encapsidated by AAV9 capsid proteins. 12. The AAV vector for use according to para 8, the use according to para 8, or the method according to para 8, wherein the AAV vector particle is not encapsidated by AAV8 capsid proteins or AAV9 capsid proteins. 13. The AAV vector for use according to any of paras 1 or 4-12, the use according to any of paras 2 or 4-12, or the method according to any of paras 3 or 4-12, wherein the transgene is a therapeutic transgene. 14. The AAV vector for use according to any of paras 1 or 4-13, the use according to any of paras 2 or 4-13, or the method according to any of paras 3 or 4-13, wherein the AAV vector does not comprise a reporter transgene, such as LacZ. 15. The AAV vector for use according to any of paras 1 or 4-14, the use according to any of paras 2 or 4-14, or the method according to any of paras 3 or 4-14, wherein the AAV vector does not comprise a nephrotic syndrome-associated transgene, such as NPHS2, ADCK4, ALG1, ARHGAP24, ARGHDIA, CD151, CD2AP, COQ2, COQ6, DGKE, E2F3, EMP2, KANK2, LAGE3, LMNA, LMX1B, MAFB, NUP85, NUP93, NXF5, OSGEP, PAX2, PDSS2, PMM2, PODXL, SCARB2, SGPL1, Smad7, TP53RK, TPRKB, VDR, WDR73, WT1, ZMPSTE24, APOL1, NPHS1, TRPC6, NUP107, NUP133, NUP160, ACTN4, INF2, ANKFY1, ANLN, CRB2, ITGA3, KANK1, KANK4, MAGI2, MYO1E, OCRL, PTPRO, SMARCAL1, SYNPO, TBC1D8B, XPO5, TNS2 or NLRP3. 16. The AAV vector for use according to any of paras 1 or 4-15, the use according to any of paras 2 or 4-15, or the method according to any of paras 3 or 4-15, wherein the AAV vector does not comprise a vascular endothelial growth factor (VEGFC) transgene. 17. The AAV vector for use according to any of paras 1 or 4-16, the use according to any of paras 2 or 4-16, or the method according to any of paras 3 or 4-16, wherein the AAV vector does not comprise a COL4A3, COL4A4, or COL4A5 transgene. 18. The AAV vector for use according to any of paras 1 or 4-17, the use according to any of paras 2 or 4-17, or the method according to any of paras 3 or 4-17, wherein the AAV vector does not comprise a nucleotide sequence encoding a complement protein, such as CFI, CFH, FHL-1, C1INH, C4BP, MASP2, C3, C5aR1, C5, C5a, CD55, CD35, CD46, CD59, vitronectin, or clusterin. 19. The AAV vector for use according to any of paras 1 or 4-18, the use according to any of paras 2 or 4-18, or the method according to any of paras 3 or 4-18, wherein the AAV vector does not comprise a NPHS2, ADCK4, ALG1, ARHGAP24, ARGHDIA, CD151, CD2AP, COQ2, COQ6, DGKE, E2F3, EMP2, KANK2, LAGE3, LMNA, LMX1B, MAFB, NUP85, NUP93, NXF5, OSGEP, PAX2, PDSS2, PMM2, PODXL, SCARB2, SGPL1, Smad7, TP53RK, TPRKB, VDR, WDR73, WT1, ZMPSTE24, APOL1, NPHS1, TRPC6, NUP107, NUP133, NUP160, ACTN4, INF2, ANKFY1, ANLN, CRB2, ITGA3, KANK1, KANK4, MAGI2, MYO1E, OCRL, PTPRO, SMARCAL1, SYNPO, TBC1D8B, XPO5, TNS2. NLRP3, VEGFC, COL4A3, COL4A4, COL4A5, CFI, CFH, FHL-1, C1INH, C4BP, MASP2, C3, C5aR1, C5, C5a, CD55, CD35, CD46, CD59, vitronectin, or clusterin transgene. 20. The AAV vector for use according to any of paras 1 or 4-19, the use according to any of paras 2 or 4-19, or the method according to any of paras 3 or 4-19, wherein the transgene is operably linked to a Woodchuck hepatitis post-transcriptional regulatory element (WPRE). 21. The AAV vector for use according to any of paras 1 or 4-20, the use according to any of paras 2 or 4-20, or the method according to any of paras 3 or 4-20, wherein the transgene is operably linked to a polyadenylation signal. 22. The AAV vector for use according to any of paras 1 or 4-21, the use according to any of paras 2 or 4-21, or the method according to any of paras 3 or 4-21, wherein the transgene is operably linked to a Kozak sequence. 23. The AAV vector for use according to any of paras 1 or 4-22, the use according to any of paras 2 or 4-22, or the method according to any of paras 3 or 4-22, wherein the expression of the transgene in the podocytes of said subject treats or prevents a kidney disease or a glomerular disease in said subject. 24. The AAV vector for use according to any of paras 1 or 4-23, the use according to any of paras 2 or 4-23, or the method according to any of paras 3 or 4-23, wherein the subject has a defective gene corresponding to said transgene. 25. The AAV vector for use according to any of paras 1 or 4-24, the use according to any of paras 2 or 4-24, or the method according to any of paras 3 or 4-24, wherein the subject is a human subject. 26. The AAV vector for use according to any of paras 1 or 4-25, the use according to any of paras 2 or 4-25, or the method according to any of paras 3 or 4-25, wherein the subject is suffering from or is at risk of a kidney disease or a glomerular disease. 27. The AAV vector for use according to any of paras 1 or 4-26, the use according to any of paras 2 or 4-26, or the method according to any of paras 3 or 4-26, wherein the AAV vector is administered in the form of a pharmaceutical composition comprising a pharmaceutically acceptable carrier, diluent or excipient. 28. The AAV vector for use according to any of paras 1 or 4-27, the use according to any of paras 2 or 4-27, or the method according to any of paras 3 or 4-27, wherein the AAV vector is administered systemically and/or by intravenous injection. 29. The AAV vector for use according to any of paras 1 or 4-28, the use according to any of paras 2 or 4-28, or the method according to any of paras 3 or 4-28, wherein the AAV vector is administered locally and/or by injection into the renal artery. 30. The AAV vector for use according to any of paras 1 or 4-29, the use according to any of paras 2 or 4-29, or the method according to any of paras 3 or 4-29, wherein the AAV vector is administered in a dose of from about 1x106 vg/kg to about 1x1014 vg/kg, from about 1x106 vg/kg to about 1x1013 vg/kg, or from about 1x109 vg/kg to about 1x1012 vg/kg. 31. The AAV vector for use according to any of paras 1 or 4-30, the use according to any of paras 2 or 4-30, or the method according to any of paras 3 or 4-30, wherein the transgene is specifically expressed in podocytes. 32. The AAV vector for use according to any of paras 1 or 4-31, the use according to any of paras 2 or 4-31, or the method according to any of paras 3 or 4-31, wherein the transgene is expressed in 5% or more podocytes in at least one of the subject’s kidneys. 33. An AAV vector comprising a therapeutic transgene, wherein the transgene is operably linked to a 5’-truncated podocin promoter, and wherein the AAV vector does not comprise: (i) a nephrotic syndrome-associated transgene, such as NPHS2, ADCK4, ALG1, ARHGAP24, ARGHDIA, CD151, CD2AP, COQ2, COQ6, DGKE, E2F3, EMP2, KANK2, LAGE3, LMNA, LMX1B, MAFB, NUP85, NUP93, NXF5, OSGEP, PAX2, PDSS2, PMM2, PODXL, SCARB2, SGPL1, Smad7, TP53RK, TPRKB, VDR, WDR73, WT1, ZMPSTE24, APOL1, NPHS1, TRPC6, NUP107, NUP133, NUP160, ACTN4, INF2, ANKFY1, ANLN, CRB2, ITGA3, KANK1, KANK4, MAGI2, MYO1E, OCRL, PTPRO, SMARCAL1, SYNPO, TBC1D8B, XPO5, TNS2 or NLRP3; (ii) a vascular endothelial growth factor (VEGFC) transgene; (iii) a COL4A3, COL4A4, or COL4A5 transgene; or (iv) a nucleotide sequence encoding a complement protein, such as CFI, CFH, FHL-1, C1INH, C4BP, MASP2, C3, C5aR1, C5, C5a, CD55, CD35, CD46, CD59, vitronectin, or clusterin. 34. An AAV vector particle encapsidated by AAV9 capsid proteins and comprising a therapeutic transgene, wherein the transgene is operably linked to a 5’-truncated podocin promoter. 35. An AAV vector particle encapsidated by LK03 capsid proteins and comprising a therapeutic transgene, wherein the transgene is operably linked to a 5’-truncated podocin promoter. 36. The AAV vector according to any of paras 33-35, wherein the 5’-truncated podocin promoter is a truncate of a full-length podocin promoter consisting of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 1. 37. The AAV vector according to any of paras 33-36, wherein the 5’-truncated podocin promoter has a length of about 1.5 kb or less, about 1.4 kb or less, about 1.3 kb or less, about 1.2 kb or less, about 1.1 kb or less, about 1.0 kb or less, about 0.9 kb or less, about 0.8 kb or less, about 0.7 kb or less, or about 0.6 kb or less. 38. The AAV vector according to any of paras 33-37, wherein the 5’-truncated podocin promoter has a length of 628 bp or less. 39. The AAV vector according to any of paras 33-38, wherein the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 2. 40. The AAV vector according to any of paras 33-39, wherein the transgene is operably linked to a Woodchuck hepatitis post-transcriptional regulatory element (WPRE). 41. The AAV vector according to any of paras 33-40, wherein the transgene is operably linked to a polyadenylation signal. 42. The AAV vector according to any of paras 33-41, wherein the transgene is operably linked to a Kozak sequence. 43. A cell comprising the AAV vector according to any of paras 33-42. 44. A pharmaceutical composition comprising the AAV vector according to any of paras 33- 42, or the cell according to para 43. 45. The AAV vector according to any of paras 33-42, the cell according to para 43, or the pharmaceutical composition according to para 44, for use in medicine, optionally for use in a method according to any of paras 3-32. 46. A promoter consisting of a nucleotide sequence having at least 97.8% identity to SEQ ID NO: 2. 47. The promoter according to para 46, wherein promoter consists of a nucleotide sequence having at least 97.9%, at least 98.0%, at least 98.1%, at least 98.2%, at least 98.3%, at least 98.4%, at least 98.5%, at least 98.6%, at least 98.7%, at least 98.8%, at least 98.9%, at least 99.0%, at least 99.1%, at least 99.2%, at least 99.3%, at least 99.4%, at least 99.5%, at least 99.6%, at least 99.7%, at least 99.8%, or at least 99.9% identity to SEQ ID NO: 2. 48. The promoter according to para 46 or 47, wherein the promoter consists of the nucleotide sequence of SEQ ID NO: 2. 49. A polynucleotide comprising the promoter according to any of paras 46-48. 50. The polynucleotide according to para 49, wherein the promoter is operably linked to a transgene. 51. The polynucleotide according to para 49 or 50, wherein the transgene is operably linked to one or more further regulatory elements, such as a post-transcriptional regulatory element and/or a polyadenylation sequence. 52. A vector comprising the polynucleotide according to any of paras 49-51. 53. The vector according to para 52, wherein the vector is capable of transducing podocytes, optionally wherein the vector is capable of specifically transducing podocytes. 54. The vector according to para 52 or 53, wherein the vector is a viral vector, such as an adeno-associated virus (AAV) vector, a lentiviral vector, a retroviral vector, an adenoviral vector, an adeno-associated viral vector, a herpes simplex viral vector, an alphaviral vector, a flaviviral vector, a rhabdoviral vector, a measles viral vector, a Newcastle disease viral vector, a poxviral vector, and a picornaviral vector, preferably wherein the vector is an AAV vector. 55. The vector according to para 54, wherein the viral vector is in the form of a viral vector particle, preferably wherein the viral vector is in the form of an AAV vector particle. 56. The vector according to any of paras 59-62, wherein the vector is the form of an AAV vector particle encapsidated by AAV3B, LK03, AAV9, ShH10, AAV-DJ, AAV2, AAV6.2, or AAV5 capsid proteins. 57. A cell comprising the polynucleotide according to any of paras 49-51, or the vector according to any of paras 52-56. 58. A pharmaceutical composition comprising the polynucleotide according to any of paras 49-51, the vector according to any of paras 52-56, or the cell according to para 57. 59. The polynucleotide according to any of paras 49-51, the vector according to any of paras 52-56, or the cell according to para 57, for use in medicine, optionally for use in a method according to any of paras 3-32. 60. Use of the promoter according to any of paras 46-48 to drive expression of a transgene. 61. The use according to para 60, wherein the expression is kidney-specific. All publications mentioned in the above specification are herein incorporated by reference. Various modifications and variations of the disclosed methods, cells, compositions and uses of the invention will be apparent to the skilled person without departing from the scope and spirit of the invention. Although the invention has been disclosed in connection with specific preferred embodiments, it should be understood that the invention as claimed should not be unduly limited to such specific embodiments. Indeed, various modifications of the disclosed modes for carrying out the invention, which are obvious to the skilled person are intended to be within the scope of the following claims.

Claims

CLAIMS 1. An AAV vector comprising a transgene for use in gene therapy, wherein said AAV vector is administered to a subject, wherein the transgene is expressed in podocytes of said subject, and wherein the transgene is operably linked to a 5’-truncated podocin promoter. 2. Use of an AAV vector comprising a transgene in the manufacture of a medicament for gene therapy, wherein said AAV vector is administered to a subject, wherein the transgene is expressed in podocytes of said subject, and wherein the transgene is operably linked to a 5’- truncated podocin promoter. 3. A method of expressing a transgene in podocytes of a subject, the method comprising administering an AAV vector comprising the transgene to said subject, wherein the transgene is expressed in podocytes of said subject, and wherein the transgene is operably linked to a 5’-truncated podocin promoter. 4. The AAV vector for use according to claim 1, the use according to claim 2, or the method according to claim 3, wherein the 5’-truncated podocin promoter is a truncate of a full-length podocin promoter consisting of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 1. 5. The AAV vector for use according to claim 1 or 4, the use according to claim 2 or 4, or the method according to claim 3 or 4, wherein the 5’-truncated podocin promoter has a length of about 1.5 kb or less, about 1.4 kb or less, about 1.3 kb or less, about 1.2 kb or less, about 1.1 kb or less, about 1.0 kb or less, about 0.9 kb or less, about 0.8 kb or less, about 0.7 kb or less, or about 0.6 kb or less. 6. The AAV vector for use according to any of claims 1 or 4-5, the use according to any of claims 2 or 4-5, or the method according to any of claims 3 or 4-5, wherein the 5’-truncated podocin promoter has a length of 628 bp or less, 600 bp or less, 550 bp or less, 500 bp or less, 450 bp or less, 400 bp or less, 350 bp or less, 300 bp or less, 250 bp or less, or 200 bp or less. 7. The AAV vector for use according to any of claims 1 or 4-6, the use according to any of claims 2 or 4-6, or the method according to any of claims 3 or 4-6, wherein the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to any of SEQ ID NOs: 2 or 35-54, optionally wherein the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 2. 8. The AAV vector for use according to any of claims 1 or 4-7, the use according to any of claims 2 or 4-7, or the method according to any of claims 3 or 4-7, wherein the AAV vector is administered to the subject in the form of an AAV vector particle. 9. The AAV vector for use according to claim 8, the use according to claim 8, or the method according to claim 8, wherein the AAV vector particle is encapsidated by AAV3B, LK03, or AAV9 capsid proteins. 10. The AAV vector for use according to claim 8, the use according to claim 8, or the method according to claim 8, wherein the AAV vector particle is encapsidated by LK03 capsid proteins. 11. The AAV vector for use according to claim 8, the use according to claim 8, or the method according to claim 8, wherein the AAV vector particle is encapsidated by AAV9 capsid proteins. 12. The AAV vector for use according to claim 8, the use according to claim 8, or the method according to claim 8, wherein the AAV vector particle is not encapsidated by AAV8 capsid proteins or AAV9 capsid proteins. 13. The AAV vector for use according to any of claims 1 or 4-12, the use according to any of claims 2 or 4-12, or the method according to any of claims 3 or 4-12, wherein the transgene is a therapeutic transgene. 14. The AAV vector for use according to any of claims 1 or 4-13, the use according to any of claims 2 or 4-13, or the method according to any of claims 3 or 4-13, wherein the AAV vector does not comprise a reporter transgene, such as LacZ. 15. The AAV vector for use according to any of claims 1 or 4-14, the use according to any of claims 2 or 4-14, or the method according to any of claims 3 or 4-14, wherein the AAV vector does not comprise a nephrotic syndrome-associated transgene, such as NPHS2, ADCK4, ALG1, ARHGAP24, ARGHDIA, CD151, CD2AP, COQ2, COQ6, DGKE, E2F3, EMP2, KANK2, LAGE3, LMNA, LMX1B, MAFB, NUP85, NUP93, NXF5, OSGEP, PAX2, PDSS2, PMM2, PODXL, SCARB2, SGPL1, Smad7, TP53RK, TPRKB, VDR, WDR73, WT1, ZMPSTE24, APOL1, NPHS1, TRPC6, NUP107, NUP133, NUP160, ACTN4, INF2, ANKFY1, ANLN, CRB2, ITGA3, KANK1, KANK4, MAGI2, MYO1E, OCRL, PTPRO, SMARCAL1, SYNPO, TBC1D8B, XPO5, TNS2 or NLRP3. 16. The AAV vector for use according to any of claims 1 or 4-15, the use according to any of claims 2 or 4-15, or the method according to any of claims 3 or 4-15, wherein the AAV vector does not comprise a vascular endothelial growth factor (VEGFC) transgene. 17. The AAV vector for use according to any of claims 1 or 4-16, the use according to any of claims 2 or 4-16, or the method according to any of claims 3 or 4-16, wherein the AAV vector does not comprise a COL4A3, COL4A4, or COL4A5 transgene. 18. The AAV vector for use according to any of claims 1 or 4-17, the use according to any of claims 2 or 4-17, or the method according to any of claims 3 or 4-17, wherein the AAV vector does not comprise a nucleotide sequence encoding a complement protein, such as CFI, CFH, FHL-1, C1INH, C4BP, MASP2, C3, C5aR1, C5, C5a, CD55, CD35, CD46, CD59, vitronectin, or clusterin. 19. The AAV vector for use according to any of claims 1 or 4-18, the use according to any of claims 2 or 4-18, or the method according to any of claims 3 or 4-18, wherein the AAV vector does not comprise a NPHS2, ADCK4, ALG1, ARHGAP24, ARGHDIA, CD151, CD2AP, COQ2, COQ6, DGKE, E2F3, EMP2, KANK2, LAGE3, LMNA, LMX1B, MAFB, NUP85, NUP93, NXF5, OSGEP, PAX2, PDSS2, PMM2, PODXL, SCARB2, SGPL1, Smad7, TP53RK, TPRKB, VDR, WDR73, WT1, ZMPSTE24, APOL1, NPHS1, TRPC6, NUP107, NUP133, NUP160, ACTN4, INF2, ANKFY1, ANLN, CRB2, ITGA3, KANK1, KANK4, MAGI2, MYO1E, OCRL, PTPRO, SMARCAL1, SYNPO, TBC1D8B, XPO5, TNS2. NLRP3, VEGFC, COL4A3, COL4A4, COL4A5, CFI, CFH, FHL-1, C1INH, C4BP, MASP2, C3, C5aR1, C5, C5a, CD55, CD35, CD46, CD59, vitronectin, or clusterin transgene. 20. The AAV vector for use according to any of claims 1 or 4-19, the use according to any of claims 2 or 4-19, or the method according to any of claims 3 or 4-19, wherein the transgene is operably linked to a Woodchuck hepatitis post-transcriptional regulatory element (WPRE). 21. The AAV vector for use according to any of claims 1 or 4-20, the use according to any of claims 2 or 4-20, or the method according to any of claims 3 or 4-20, wherein the transgene is operably linked to a polyadenylation signal. 22. The AAV vector for use according to any of claims 1 or 4-21, the use according to any of claims 2 or 4-21, or the method according to any of claims 3 or 4-21, wherein the transgene is operably linked to a Kozak sequence.
23. The AAV vector for use according to any of claims 1 or 4-22, the use according to any of claims 2 or 4-22, or the method according to any of claims 3 or 4-22, wherein the expression of the transgene in the podocytes of said subject treats or prevents a kidney disease or a glomerular disease in said subject. 24. The AAV vector for use according to any of claims 1 or 4-23, the use according to any of claims 2 or 4-23, or the method according to any of claims 3 or 4-23, wherein the subject has a defective gene corresponding to said transgene. 25. The AAV vector for use according to any of claims 1 or 4-24, the use according to any of claims 2 or 4-24, or the method according to any of claims 3 or 4-24, wherein the subject is a human subject. 26. The AAV vector for use according to any of claims 1 or 4-25, the use according to any of claims 2 or 4-25, or the method according to any of claims 3 or 4-25, wherein the subject is suffering from or is at risk of a kidney disease or a glomerular disease. 27. The AAV vector for use according to any of claims 1 or 4-26, the use according to any of claims 2 or 4-26, or the method according to any of claims 3 or 4-26, wherein the AAV vector is administered in the form of a pharmaceutical composition comprising a pharmaceutically acceptable carrier, diluent or excipient. 28. The AAV vector for use according to any of claims 1 or 4-27, the use according to any of claims 2 or 4-27, or the method according to any of claims 3 or 4-27, wherein the AAV vector is administered systemically and/or by intravenous injection. 29. The AAV vector for use according to any of claims 1 or 4-28, the use according to any of claims 2 or 4-28, or the method according to any of claims 3 or 4-28, wherein the AAV vector is administered locally and/or by injection into the renal artery. 30. The AAV vector for use according to any of claims 1 or 4-29, the use according to any of claims 2 or 4-29, or the method according to any of claims 3 or 4-29, wherein the AAV vector is administered in a dose of from about 1x106 vg/kg to about 1x1014 vg/kg, from about 1x106 vg/kg to about 1x1013 vg/kg, or from about 1x109 vg/kg to about 1x1012 vg/kg. 31. The AAV vector for use according to any of claims 1 or 4-30, the use according to any of claims 2 or 4-30, or the method according to any of claims 3 or 4-30, wherein the transgene is specifically expressed in podocytes.
32. The AAV vector for use according to any of claims 1 or 4-31, the use according to any of claims 2 or 4-31, or the method according to any of claims 3 or 4-31, wherein the transgene is expressed in 5% or more podocytes in at least one of the subject’s kidneys. 33. An AAV vector comprising a therapeutic transgene, wherein the transgene is operably linked to a 5’-truncated podocin promoter, and wherein the AAV vector does not comprise: (i) a nephrotic syndrome-associated transgene, such as NPHS2, ADCK4, ALG1, ARHGAP24, ARGHDIA, CD151, CD2AP, COQ2, COQ6, DGKE, E2F3, EMP2, KANK2, LAGE3, LMNA, LMX1B, MAFB, NUP85, NUP93, NXF5, OSGEP, PAX2, PDSS2, PMM2, PODXL, SCARB2, SGPL1, Smad7, TP53RK, TPRKB, VDR, WDR73, WT1, ZMPSTE24, APOL1, NPHS1, TRPC6, NUP107, NUP133, NUP160, ACTN4, INF2, ANKFY1, ANLN, CRB2, ITGA3, KANK1, KANK4, MAGI2, MYO1E, OCRL, PTPRO, SMARCAL1, SYNPO, TBC1D8B, XPO5, TNS2 or NLRP3; (ii) a vascular endothelial growth factor (VEGFC) transgene; (iii) a COL4A3, COL4A4, or COL4A5 transgene; or (iv) a nucleotide sequence encoding a complement protein, such as CFI, CFH, FHL-1, C1INH, C4BP, MASP2, C3, C5aR1, C5, C5a, CD55, CD35, CD46, CD59, vitronectin, or clusterin. 34. An AAV vector particle encapsidated by AAV9 capsid proteins and comprising a therapeutic transgene, wherein the transgene is operably linked to a 5’-truncated podocin promoter. 35. An AAV vector particle encapsidated by LK03 capsid proteins and comprising a therapeutic transgene, wherein the transgene is operably linked to a 5’-truncated podocin promoter. 36. The AAV vector according to any of claims 33-35, wherein the 5’-truncated podocin promoter is a truncate of a full-length podocin promoter consisting of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 1. 37. The AAV vector according to any of claims 33-36, wherein the 5’-truncated podocin promoter has a length of about 1.5 kb or less, about 1.4 kb or less, about 1.3 kb or less, about
1.2 kb or less, about 1.1 kb or less, about 1.0 kb or less, about 0.9 kb or less, about 0.8 kb or less, about 0.7 kb or less, or about 0.6 kb or less. 38. The AAV vector according to any of claims 33-37, wherein the 5’-truncated podocin promoter has a length of 628 bp or less, 600 bp or less, 550 bp or less, 500 bp or less, 450 bp or less, 400 bp or less, 350 bp or less, 300 bp or less, 250 bp or less, or 200 bp or less. 39. The AAV vector according to any of claims 33-38, wherein the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to any of SEQ ID NOs: 2 or 35-54, optionally wherein the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 2. 40. The AAV vector according to any of claims 33-39, wherein the transgene is operably linked to a Woodchuck hepatitis post-transcriptional regulatory element (WPRE). 41. The AAV vector according to any of claims 33-40, wherein the transgene is operably linked to a polyadenylation signal. 42. The AAV vector according to any of claims 33-41, wherein the transgene is operably linked to a Kozak sequence. 43. A cell comprising the AAV vector according to any of claims 33-42. 44. A pharmaceutical composition comprising the AAV vector according to any of claims 33- 42, or the cell according to claim 43. 45. The AAV vector according to any of claims 33-42, the cell according to claim 43, or the pharmaceutical composition according to claim 44, for use in medicine, optionally for use in a method according to any of claims 3-32. 46. A promoter consisting of a nucleotide sequence having at least 97.8% identity to SEQ ID NO: 2. 47. The promoter according to claim 46, wherein promoter consists of a nucleotide sequence having at least 97.9%, at least 98.0%, at least 98.1%, at least 98.2%, at least 98.3%, at least 98.4%, at least 98.5%, at least 98.6%, at least 98.7%, at least 98.8%, at least 98.9%, at least
99.0%, at least 99.1%, at least 99.2%, at least 99.3%, at least 99.4%, at least 99.5%, at least 99.6%, at least 99.7%, at least 99.8%, or at least 99.9% identity to SEQ ID NO: 2. 48. The promoter according to claim 46 or 47, wherein the promoter consists of the nucleotide sequence of SEQ ID NO: 2. 49. A 5’-truncated podocin promoter having a length of 600bp or less. 50. The promoter according to claim 49, wherein the full-length podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 1. 51. The promoter according to claim 49 or 50, wherein the 5’-truncated podocin promoter has a length of 550bp or less, 500bp or less, 450bp or less, 400bp or less, 350 bp or less, 300 bp or less, 250 bp or less, or 200 bp or less. 52. The promoter according to any of claims 49-51, wherein the 5’-truncated podocin promoter consists of a nucleotide sequence having at least 70%, at least 75%, at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to any of SEQ ID NOs: 36-54. 53. A promoter which consists of a nucleotide sequence having at least 90% identity to any of SEQ ID NOs: 36-54. 54. The promoter according to claim 53, wherein the promoter consists of a nucleotide sequence having at least 90% identity to any of SEQ ID NOs: 37-46 or 47-54, any of SEQ ID NOs: 37-46 or 48-54, any of SEQ ID NOs: 38-46 or 48-54, any of SEQ ID NOs: 38-46 or 49- 54, any of SEQ ID NOs: 39-46 or 49-54, any of SEQ ID NOs: 39-46 or 50-54, any of SEQ ID NOs: 40-46 or 50-54, any of SEQ ID NOs: 40-46 or 51-54, any of SEQ ID NOs: 41-46 or 51- 54, any of SEQ ID NOs: 41-46 or 52-54, any of SEQ ID NOs: 42-46 or 52-54, any of SEQ ID NOs: 42-46 or 53-54, any of SEQ ID NOs: 43-46 or 53-54, or any of SEQ ID NOs: 43-46 or 54. 55. The promoter according to claim 53, wherein the promoter consists of a nucleotide sequence having at least 90% identity to any of SEQ ID NOs: 38, 40, 41, 42, 44, 47, 48, 51 or 53, preferably wherein the promoter consists of a nucleotide sequence having at least 90% identity to any of SEQ ID NOs: 40, 47 or 53.
56. The promoter according to claim 53, wherein the promoter consists of a nucleotide sequence having at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 36. 57. The promoter according to claim 53, wherein the promoter consists of a nucleotide sequence having at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 47. 58. The promoter according to claim 53, wherein the promoter consists of a nucleotide sequence having at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 37. 59. The promoter according to claim 53, wherein the promoter consists of a nucleotide sequence having at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 48. 60. The promoter according to claim 53, wherein the promoter consists of a nucleotide sequence having at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 38. 61. The promoter according to claim 53, wherein the promoter consists of a nucleotide sequence having at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 49. 62. The promoter according to claim 53, wherein the promoter consists of a nucleotide sequence having at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 39. 63. The promoter according to claim 53, wherein the promoter consists of a nucleotide sequence having at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 50.
64. The promoter according to claim 53, wherein the promoter consists of a nucleotide sequence having at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 40. 65. The promoter according to claim 53, wherein the promoter consists of a nucleotide sequence having at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 51. 66. The promoter according to claim 53, wherein the promoter consists of a nucleotide sequence having at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 41. 67. The promoter according to claim 53, wherein the promoter consists of a nucleotide sequence having at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 52. 68. The promoter according to claim 53, wherein the promoter consists of a nucleotide sequence having at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 42. 69. The promoter according to claim 53, wherein the promoter consists of a nucleotide sequence having at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 53. 70. The promoter according to claim 53, wherein the promoter consists of a nucleotide sequence having at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 43. 71. The promoter according to claim 53, wherein the promoter consists of a nucleotide sequence having at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 54.
72. The promoter according to claim 53, wherein the promoter consists of a nucleotide sequence having at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 44. 73. The promoter according to claim 53, wherein the promoter consists of a nucleotide sequence having at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 45. 74. The promoter according to claim 53, wherein the promoter consists of a nucleotide sequence having at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identity to SEQ ID NO: 46. 75. A polynucleotide comprising the promoter according to any of claims 46-74. 76. The polynucleotide according to claim 75, wherein the promoter is operably linked to a transgene. 77. The polynucleotide according to claim 75 or 76, wherein the transgene is operably linked to one or more further regulatory elements, such as a post-transcriptional regulatory element and/or a polyadenylation sequence. 78. A vector comprising the polynucleotide according to any of claims 75-77. 79. The vector according to claim 78, wherein the vector is capable of transducing podocytes, optionally wherein the vector is capable of specifically transducing podocytes. 80. The vector according to claim 78 or 79, wherein the vector is a viral vector, such as an adeno-associated virus (AAV) vector, a lentiviral vector, a retroviral vector, an adenoviral vector, an adeno-associated viral vector, a herpes simplex viral vector, an alphaviral vector, a flaviviral vector, a rhabdoviral vector, a measles viral vector, a Newcastle disease viral vector, a poxviral vector, and a picornaviral vector, preferably wherein the vector is an AAV vector. 81. The vector according to claim 80, wherein the viral vector is in the form of a viral vector particle, preferably wherein the viral vector is in the form of an AAV vector particle. 82. The vector according to any of claims 78-81, wherein the vector is the form of an AAV vector particle encapsidated by AAV3B, LK03, AAV9, ShH10, AAV-DJ, AAV2, AAV6.2, or AAV5 capsid proteins.
83. A cell comprising the polynucleotide according to any of claims 75-77, or the vector according to any of claims 78-82. 84. A pharmaceutical composition comprising the polynucleotide according to any of claims 75-77, the vector according to any of claims 78-82, or the cell according to claim 83. 85. The polynucleotide according to any of claims 75-77, the vector according to any of claims 78-82, or the cell according to claim 83, for use in medicine, optionally for use in a method according to any of claims 3-32. 86. Use of the promoter according to any of claims 46-74 to drive expression of a transgene. 87. The use according to claim 86, wherein the expression is kidney-specific.
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