WO2018232055A1 - Aadc polynucleotides for the treatment of parkinson's disease - Google Patents

Aadc polynucleotides for the treatment of parkinson's disease Download PDF

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Publication number
WO2018232055A1
WO2018232055A1 PCT/US2018/037437 US2018037437W WO2018232055A1 WO 2018232055 A1 WO2018232055 A1 WO 2018232055A1 US 2018037437 W US2018037437 W US 2018037437W WO 2018232055 A1 WO2018232055 A1 WO 2018232055A1
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seq
aav
region
aavhu
aavrh
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English (en)
French (fr)
Inventor
Maria SCHEEL
Bernard RAVINA
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Voyager Therapeutics Inc
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Voyager Therapeutics Inc
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Priority to MX2019014974A priority Critical patent/MX2019014974A/es
Priority to EA201992604A priority patent/EA201992604A1/ru
Priority to PE2019002526A priority patent/PE20200494A1/es
Priority to CA3065471A priority patent/CA3065471A1/en
Priority to BR112019025122-7A priority patent/BR112019025122A2/pt
Priority to EP18816860.3A priority patent/EP3638315A4/en
Priority to SG11201912154XA priority patent/SG11201912154XA/en
Priority to KR1020197036981A priority patent/KR20200018455A/ko
Priority to AU2018284960A priority patent/AU2018284960B2/en
Priority to JP2019569267A priority patent/JP2020527333A/ja
Priority to CN201880052382.9A priority patent/CN111107879A/zh
Application filed by Voyager Therapeutics Inc filed Critical Voyager Therapeutics Inc
Priority to US16/184,466 priority patent/US20190060425A1/en
Publication of WO2018232055A1 publication Critical patent/WO2018232055A1/en
Priority to US16/523,567 priority patent/US11759506B2/en
Priority to IL270821A priority patent/IL270821A/en
Priority to CONC2019/0013707A priority patent/CO2019013707A2/es
Priority to PH12019502793A priority patent/PH12019502793A1/en
Anticipated expiration legal-status Critical
Priority to AU2021202378A priority patent/AU2021202378A1/en
Ceased legal-status Critical Current

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    • A—HUMAN NECESSITIES
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    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
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    • 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
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    • C12N15/86—Viral vectors
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
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    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
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    • 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
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    • C12Y401/00—Carbon-carbon lyases (4.1)
    • C12Y401/01—Carboxy-lyases (4.1.1)
    • C12Y401/01028—Aromatic-L-amino-acid decarboxylase (4.1.1.28), i.e. tryptophane-decarboxylase

Definitions

  • the invention relates to compositions, particularly nucleic acid molecules, e.g., polynucleotides encoding AADC, for use in the treatment of Parkinson's disease.
  • AADC polynucleotides may be encoded by or within recombinant adeno- associated viruses (AAVs).
  • AAVs adeno- associated viruses
  • Aromatic L-amino acid decarboxylase is a homodimeric pyridoxal phosphate-dependent enzyme responsible for the synthesis of dopamine and serotonin.
  • the encoded protein catalyzes the decarboxylation of L-3,4-dihydroxyphenylalanine (L-DOPA or levodopa) to dopamine; L-5-hydroxytryptophan to serotonin; and L-tryptophan to tryptamine.
  • AADCD aromatic L-amino-acid decarboxylase deficiency
  • Parkinson's Disease is a progressive neurodegenerative disease of the central nervous system (CNS) producing sensory and motor symptoms.
  • Dopamine replacement i.e., levodopa
  • the benefit of dopamine therapy becomes less marked over time, due, in part, to the progressive death of dopamine-generating cells and corresponding loss of AADC activity.
  • systemic administration of high-dose dopamine is complicated by side effects, such as fluctuations in motor performance, dyskinesias, and hallucinations, resulting from dopaminergic stimulation of the mesolimbic system.
  • One strategy to restore dopaminergic function and minimize side effects is the use of gene therapy to deliver AADC directly to a targeted region of the CNS.
  • AAV adeno-associated virus
  • the present invention provides such improved nucleic acid constructs, e.g.,
  • polynucleotides for use with AAV-derived vectors comprising dopa carboxylase (“DDC") gene sequence which encodes a full-length AADC protein for the purpose of gene therapy in the treatment of Parkinson's Disease.
  • DDC dopa carboxylase
  • nucleic acid constructs described herein comprise at least a 5'-ITR and a 3'-ITR, each or both of which may be derived from an AAV, positioned about a DDC gene sequence, as well as additional components required for gene expression and clone selection.
  • compositions, methods, processes, kits and devices for the design, preparation, manufacture and/or formulation of AADC polynucleotides are Described herein.
  • such AADC polynucleotides may be encoded by or contained within plasmids or vectors or recombinant adeno-associated viruses (AAV).
  • AAV adeno-associated viruses
  • FIG. 1 is a schematic of a viral genome of the invention.
  • AAVs Adeno-associated viruses
  • AAV particles Adeno-associated viruses
  • Viruses of the Parvoviridae family are small non-enveloped icosahedral capsid viruses characterized by a single stranded DNA genome. Parvoviridae family viruses consist of two subfamilies: Parvovirinae, which infect vertebrates, and Densovirinae, which infect invertebrates. Due to its relatively simple structure, easily manipulated using standard molecular biology techniques, this virus family is useful as a biological tool.
  • the genome of the virus may be modified to contain a minimum of components for the assembly of a functional recombinant virus, or viral particle, which is loaded with or engineered to express or deliver a desired payload, which may be delivered to a target cell, tissue, organ, or organism.
  • parvoviruses and other members of the Parvoviridae family are generally described in Kenneth I. Berns, "Parvoviridae: The Viruses and Their Replication," Chapter 69 in FIELDS VIROLOGY (3d Ed. 1996), the contents of which are incorporated by reference in their entirety.
  • the Parvoviridae family comprises the Dependovirus genus which includes adeno- associated viruses (AAV) capable of replication in vertebrate hosts including, but not limited to, human, primate, bovine, canine, equine, and ovine species.
  • AAV adeno- associated viruses
  • the vector genome is a linear, single-stranded DNA (ssDNA) molecule approximately 5,000 nucleotides (nt) in length.
  • the AAV viral genome can comprise a payload region and at least one inverted terminal repeat (ITR) or ITR region. ITRs traditionally flank the coding nucleotide sequences for the non- structural proteins (encoded by Rep genes) and the structural proteins (encoded by capsid genes or Cap genes). While not wishing to be bound by theory, an AAV viral genome typically comprises two ITR sequences.
  • the vector genome comprises a characteristic T- shaped hairpin structure defined by the self-complementary terminal 145 nt of the 5' and 3' ends of the ssDNA which form an energetically stable double stranded region.
  • the double stranded hairpin structures comprise multiple functions including, but not limited to, acting as an origin for DNA replication by functioning as primers for the endogenous DNA polymerase complex of the host viral replication cell.
  • AAV particles may comprise the viral genome, in whole or in part, of any naturally occurring and/or recombinant AAV serotype nucleotide sequence or variant.
  • AAV variants may have sequences of significant homology at the nucleic acid (genome or capsid) and amino acid levels (capsids), to produce constructs which are generally physical and functional equivalents, replicate by similar mechanisms, and assemble by similar mechanisms. Chiorini et al., J. Vir. 71 : 6823-33(1997); Srivastava et al., J. Vir. 45:555-64 (1983); Chiorini et al., J. Vir.
  • AAV particles of the present invention are recombinant AAV particles which are replication defective, lacking sequences encoding functional Rep and Cap proteins within their viral genome. These defective AAV particles may lack most or all parental coding sequences and essentially carry only one or two AAV ITR sequences and the nucleic acid of interest for delivery to a cell, a tissue, an organ or an organism.
  • the viral genome of the AAV particles of the present invention comprise at least one control element which provides for the replication, transcription and translation of a coding sequence encoded therein. Not all of the control elements need always be present as long as the coding sequence is capable of being replicated, transcribed and/or translated in an appropriate host cell.
  • expression control elements include sequences for transcription initiation and/or termination, promoter and/or enhancer sequences, efficient RNA processing signals such as splicing and polyadenylation signals, sequences that stabilize cytoplasmic mRNA, sequences that enhance translation efficacy (e.g., Kozak consensus sequence), sequences that enhance protein stability, and/or sequences that enhance protein processing and/or secretion.
  • AAV particles for use in therapeutics and/or diagnostics comprise a virus that has been distilled or reduced to the minimum components necessary for transduction of a nucleic acid payload or cargo of interest.
  • AAV particles are engineered as vehicles for specific delivery while lacking the deleterious replication and/or integration features found in wild-type viruses.
  • AAV particles of the present invention may be produced recombinantly and may be based on adeno-associated virus (AAV) parent or reference sequences.
  • AAV adeno-associated virus
  • a "vector” is any molecule or moiety which transports, transduces or otherwise acts as a carrier of a heterologous molecule such as the nucleic acids described herein.
  • scAAV particles contain DNA strands which anneal together to form double stranded DNA. By skipping second strand synthesis, scAAVs allow for rapid expression in the cell.
  • the AAV particle of the present invention is an scAAV.
  • the AAV particle of the present invention is an ssAAV.
  • AAV particles may be modified to enhance the efficiency of delivery. Such modified AAV particles can be packaged efficiently and be used to successfully infect the target cells at high frequency and with minimal toxicity.
  • the capsids of the AAV particles are engineered according to the methods described in US Publication Number US 20130195801, the contents of which are incorporated herein by reference in their entirety.
  • the AAV particles comprising a payload region encoding the polypeptides of the invention may be introduced into mammalian cells.
  • AAV particles of the present invention may comprise or be derived from any natural or recombinant AAV serotype. According to the present invention, the AAV particles may utilize or be based on a serotype selected from any of the following PHP.B, PHP. A, AAV1, AAV2,
  • AAV16.8/hu. lO AAV16.12/hu. l l, AAV29.3/bb. l, AAV29.5/bb.2, AAV106.1/hu.37,
  • AAV-PAEC AAV-LK01, AAV-LK02, AAV-LK03, AAV-LK04, AAV-LK05, AAV-LK06, AAV- LK07, AAV-LK08, AAV-LK09, AAV-LKIO, AAV-LKl l, AAV-LK12, AAV-LK13, AAV-LK14, AAV-LK15, AAV-LK16, AAV-LK17, AAV-LK18, AAV-LK19, AAV-PAEC2,
  • AAV128.1/hu.43 true type AAV (ttAAV), UPENN AAV 10, Japanese AAV 10 serotypes, AAV CBr-7.1, AAV CBr-7.10, AAV CBr-7.2, AAV CBr-7.3, AAV CBr-7.4, AAV CBr-7.5, AAV CBr-7.7, AAV CBr-7.8, AAV CBr-B7.3, AAV CBr-B7.4, AAV CBr-El, AAV CBr-E2, AAV CBr-E3, AAV CBr-E4,
  • the AAV serotype may be, or have, a modification as described in United States Publication No. US 20160361439, the contents of which are herein incorporated by reference in their entirety, such as but not limited to, Y252F, Y272F, Y444F, Y500F, Y700F, Y704F, Y730F, Y275F, Y281F, Y508F, Y576F, Y612G, Y673F, and Y720F of the wild-type AAVl, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV10, AAV11, AAV 12, and hybrids thereof.
  • the AAV serotype may be, or have, a mutation as described in United States Patent No. US 9546112, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, at least two, but not all the F129L, D418E, K53 IE, L584F, V598A and H642N mutations in the sequence of AAV6 (SEQ ID NO:4 of US 9546112), AAVl (SEQ ID NO:6 of US 9546112), AAV2, AAV3, AAV4, AAV5, AAV7, AAV9, AAV10 or AAVl 1 or derivatives thereof.
  • the AAV serotype may be, or have, an AAV6 sequence comprising the K53 IE mutation (SEQ ID NO:5 of US 95461 12).
  • the AAV serotype may be, or have, a mutation in the AAVl sequence, as described in in United States Publication No. US 20130224836, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, at least one of the surface-exposed tyrosine residues, preferably, at positions 252, 273, 445, 701, 705 and 731 of AAVl (SEQ ID NO: 2 of US 20130224836) substituted with another amino acid, preferably with a phenylalanine residue.
  • a mutation in the AAVl sequence as described in in United States Publication No. US 20130224836, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, at least one of the surface-exposed tyrosine residues, preferably, at positions 252, 273, 445, 701, 705 and 731 of AAVl (SEQ ID NO: 2 of US 20130224836) substituted with another amino acid, preferably with a pheny
  • the AAV serotype may be, or have, a mutation in the AAV9 sequence, such as, but not limited to, at least one of the surface-exposed tyrosine residues, preferably, at positions 252, 272, 444, 500, 700, 704 and 730 of AAV2 (SEQ ID NO: 4 of US 20130224836) substituted with another amino acid, preferably with a phenylalanine residue.
  • the tyrosine residue at position 446 of AAV9 (SEQ ID NO: 6 US 20130224836) is substituted with a phenylalanine residue.
  • the serotype may be AAV2 or a variant thereof, as described in International Publication No. WO2016130589, herein incorporated by reference in its entirety.
  • the amino acid sequence of AAV2 may comprise N587A, E548A, or N708A mutations.
  • the amino acid sequence of any AAV may comprise a V708K mutation.
  • the AAV serotype may be, or have, a sequence as described in United States Publication No. US20030138772, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, AAVl (SEQ ID NO: 6 and 64 of US20030138772), AAV2 (SEQ ID NO: 7 and 70 of US20030138772), AAV3 (SEQ ID NO: 8 and 71 of US20030138772), AAV4 (SEQ ID NO: 63 of US20030138772), AAV5 (SEQ ID NO: 114 of US20030138772), AAV6 (SEQ ID NO: 65 of US20030138772), AAV7 (SEQ ID NO: 1-3 of US20030138772), AAV8 (SEQ ID NO: 4 and 95 of US20030138772), AAV9 (SEQ ID NO: 5 and 100 of US20030138772), AAV10 (SEQ ID NO: 117 of US20030138772, the contents of which are herein
  • the AAV serotype may be, or have, a sequence as described in United States Publication No. US20150159173, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, AAV2 (SEQ ID NO: 7 and 23 of
  • rh.58 SEQ ID NO: 48 of US20150159173), rh.58 (SEQ ID NO: 48 of US20150159173), or variants thereof including, but not limited to Cy5Rl, Cy5R2, Cy5R3, Cy5R4, rh. l3R, rh.37R2, rh.2R, rh.8R, rh.48.1, rh.48.2, rh.48.1.2, hu.44Rl, hu.44R2, hu.44R3, hu.29R, ch.5Rl, rh64Rl, rh64R2, AAV6.2, AAV6.1, AAV6.12, hu.48Rl, hu.48R2, and hu.48R3.
  • the AAV serotype may be, or have, a sequence as described in United States Patent No. US 7198951, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, AAV9 (SEQ ID NO: 1-3 of US 7198951), AAV2 (SEQ ID NO: 4 of US 7198951), AAV1 (SEQ ID NO: 5 of US 7198951), AAV3 (SEQ ID NO: 6 of US 7198951), and AAV8 (SEQ ID NO: 7 of US7198951). [00036] In some embodiments, the AAV serotype may be, or have, a mutation in the AAV9 sequence as described by N Pulichla et al.
  • the AAV serotype may be, or have, a sequence as described in United States Patent No. US 6156303, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, AAV3B (SEQ ID NO: 1 and 10 of US 6156303), AAV6 (SEQ ID NO: 2, 7 and 11 of US 6156303), AAV2 (SEQ ID NO: 3 and 8 of US 6156303), AAV3A (SEQ ID NO: 4 and 9, of US 6156303), or derivatives thereof.
  • AAV3B SEQ ID NO: 1 and 10 of US 6156303
  • AAV6 SEQ ID NO: 2, 7 and 11 of US 6156303
  • AAV2 SEQ ID NO: 3 and 8 of US 6156303
  • AAV3A SEQ ID NO: 4 and 9, of US 6156303
  • the AAV serotype may be, or have, a sequence as described in United States Publication No. US20140359799, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, AAV8 (SEQ ID NO: 1 of US20140359799), AAVDJ (SEQ ID NO: 2 and 3 of US20140359799), or variants thereof.
  • the serotype may be AAVDJ or a variant thereof, such as AAVDJ8 (or AAV-DJ8), as described by Grimm et al. (Journal of Virology 82(12): 5887-5911 (2008), herein incorporated by reference in its entirety).
  • the amino acid sequence of AAVDJ8 may comprise two or more mutations in order to remove the heparin binding domain (HBD).
  • HBD heparin binding domain
  • 7,588,772 may comprise two mutations: (1) R587Q where arginine (R; Arg) at amino acid 587 is changed to glutamine (Q; Gin) and (2) R590T where arginine (R; Arg) at amino acid 590 is changed to threonine (T; Thr).
  • K406R where lysine (K; Lys) at amino acid 406 is changed to arginine (R; Arg)
  • R587Q where arginine (R; Arg) at amino acid 587 is changed to glutamine (Q; Gin)
  • R590T where arginine (R; Arg) at amino acid 590 is changed to threonine (T; Thr).
  • the AAV serotype may be, or have, a sequence of AAV4 as described in International Publication No. WO 1998011244, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to AAV4 (SEQ ID NO: 1-20 of WO1998011244).
  • the AAV serotype may be, or have, a mutation in the AAV2 sequence to generate AAV2G9 as described in International Publication No. WO2014144229 and herein incorporated by reference in its entirety.
  • the AAV serotype may be, or have, a sequence as described in International Publication No. WO2005033321, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to AAV3-3 (SEQ ID NO: 217 of
  • WO2005033321 AAV1 (SEQ ID NO: 219 and 202 of WO2005033321), AAV106.1/hu.37 (SEQ ID No: 10 of WO2005033321), AAV114.3/hu.40 (SEQ ID No: 11 of WO2005033321),
  • AAV127.2/hu.41 (SEQ ID NO:6 and 8 of WO2005033321), AAV128.3/hu.44 (SEQ ID No: 81 of WO2005033321), AAV130.4/hu.48 (SEQ ID NO: 78 of WO2005033321), AAV145.1/hu.53 (SEQ ID No: 176 and 177 of WO2005033321), AAV145.6/hu.56 (SEQ ID NO: 168 and 192 of
  • WO2005033321 AAV16.12/hu. l l (SEQ ID NO: : 153 and 57 of WO2005033321),
  • AAV16.8/hu. lO (SEQ ID NO: : 156 and 56 of WO2005033321), AAV161.10/hu.60 (SEQ ID No: 170 of WO2005033321), AAV161.6/hu.61 (SEQ ID No: 174 of WO2005033321), AAVl-7/rh.48 (SEQ ID NO: 32 of WO2005033321), AAVl-8/rh.49 (SEQ ID NOs: 103 and 25 of
  • WO2005033321 AAV2 (SEQ ID NO: 211 and 221 of WO2005033321), AAV2-15/rh.62 (SEQ ID No: 33 and 114 of WO2005033321), AAV2-3/rh.61 (SEQ ID NO: 21 of WO2005033321), AAV2- 4/rh.50 (SEQ ID No: 23 and 108 of WO2005033321), AAV2-5/rh.51 (SEQ ID NO: 104 and 22 of WO2005033321), AAV3.1/hu.6 (SEQ ID NO: 5 and 84 of WO2005033321), AAV3.1/hu.9 (SEQ ID NO: 155 and 58 of WO2005033321), AAV3-1 l/rh.53 (SEQ ID NO: 186 and 176 of
  • WO2005033321 AAV3-3 (SEQ ID NO: 200 of WO2005033321), AAV33.12/hu. l7 (SEQ ID NO:4 of WO2005033321), AAV33.4/hu. l5 (SEQ ID No: 50 of WO2005033321), AAV33.8/hu.
  • WO2005033321 AAV58.2/hu.25 (SEQ ID No: 49 of WO2005033321), AAV6 (SEQ ID NO: 203 and 220 of WO2005033321), AAV7 (SEQ ID NO: 222 and 213 of WO2005033321), AAV7.3/hu.7 (SEQ ID No: 55 of WO2005033321), AAV8 (SEQ ID NO: 223 and 214 of WO2005033321), AAVH-l/hu. l (SEQ ID No: 46 of WO2005033321), AAVH-5/hu.3 (SEQ ID No: 44 of WO2005033321), AAVhu.
  • AAVhu. l4/AAV9 (SEQ ID NO: 123 and 3 of WO2005033321), AAVhu.15 (SEQ ID NO: 147 of WO2005033321), AAVhu.16 (SEQ ID NO: 148 of WO2005033321), AAVhu.17 (SEQ ID NO: 83 of WO2005033321), AAVhu.18 (SEQ ID NO: 149 of WO2005033321), AAVhu.19 (SEQ ID NO: 133 of WO2005033321), AAVhu.2 (SEQ ID NO: 143 of WO2005033321), AAVhu.20 (SEQ ID NO: 134 of WO2005033321), AAVhu.21 (SEQ ID NO: 135 of WO2005033321), AAVhu.22 (SEQ ID NO: 138 of WO2005033321), AAVhu.23.2 (SEQ ID NO: 137 of WO2005033321), AAVh
  • WO2005033321 AAVhu.3 (SEQ ID NO: 145 of WO2005033321), AAVhu.31 (SEQ ID NO: 121 of WO2005033321), AAVhu.32 (SEQ ID NO: 122 of WO2005033321), AAVhu.34 (SEQ ID NO: 125 of WO2005033321), AAVhu.35 (SEQ ID NO: 164 of WO2005033321), AAVhu.37 (SEQ ID NO: 88 of WO2005033321), AAVhu.39 (SEQ ID NO: 102 of WO2005033321), AAVhu.4 (SEQ ID NO: 141 of WO2005033321), AAVhu.40 (SEQ ID NO: 87 of WO2005033321), AAVhu.41 (SEQ ID NO: 91 of WO2005033321), AAVhu.42 (SEQ ID NO: 85 of WO2005033321), AAVhu.43 (
  • WO2005033321 AAVhu.49 (SEQ ID NO: 189 of WO2005033321), AAVhu.51 (SEQ ID NO: 190 of WO2005033321), AAVhu.52 (SEQ ID NO: 191 of WO2005033321), AAVhu.53 (SEQ ID NO: 186 of WO2005033321), AAVhu.54 (SEQ ID NO: 188 of WO2005033321), AAVhu.55 (SEQ ID NO: 187 of WO2005033321), AAVhu.56 (SEQ ID NO: 192 of WO2005033321), AAVhu.57 (SEQ ID NO: 193 of WO2005033321), AAVhu.58 (SEQ ID NO: 194 of WO2005033321), AAVhu.6 (SEQ ID NO: 84 of WO2005033321), AAVhu.60 (SEQ ID NO: 184 of WO2005033321),
  • AAVhu.61 (SEQ ID NO: 185 of WO2005033321), AAVhu.63 (SEQ ID NO: 195 of
  • WO2005033321 AAVhu.64 (SEQ ID NO: 196 of WO2005033321), AAVhu.66 (SEQ ID NO: 197 of WO2005033321), AAVhu.67 (SEQ ID NO: 198 of WO2005033321), AAVhu.7 (SEQ ID NO: 150 of WO2005033321), AAVhu.8 (WO2005033321 SEQ ID NO: 12), AAVhu.9 (SEQ ID NO: (SEQ ID NO: 86 of WO2005033321), AAVLG-4/rh.38 (SEQ ID No: 7 of WO2005033321), AAVN721-8/rh.43 (SEQ ID NO: 163 of WO2005033321), AAVN721-8/rh.43 (SEQ ID No: 43 of WO2005033321), AAVpi.
  • WO2005033321 WO2005033321
  • AAVrh.40 SEQ ID NO: 92 of WO2005033321
  • AAVrh.43 SEQ ID NO: 163 of WO2005033321
  • AAVrh.44 WO2005033321 SEQ ID NO: 34
  • AAVrh.45 WO2005033321 SEQ ID NO: 41
  • AAVrh.47 WO2005033321 SEQ ID NO: 38
  • AAVrh.48 SEQ ID NO: 115 of WO2005033321
  • AAVrh.49 SEQ ID NO: 103 of WO2005033321
  • AAVrh.50 SEQ ID NO: 108 of WO2005033321
  • AAVrh.51 SEQ ID NO: 104 of WO2005033321
  • AAVrh.52 SEQ ID NO: 96 of WO2005033321
  • AAVrh.53 SEQ ID NO: 97 of WO2005033321
  • AAVrh.59 (WO2005033321 SEQ ID NO: 42), AAVrh.60 (WO2005033321 SEQ ID NO: 31), AAVrh.61 (SEQ ID NO: 107 of WO2005033321), AAVrh.62 (SEQ ID NO: 114 of
  • WO2005033321 WO2005033321
  • AAVrh.64 SEQ ID NO: 99 of WO2005033321
  • AAVrh.65 WO2005033321 SEQ ID NO: 35
  • AAVrh.68 WO2005033321 SEQ ID NO: 16
  • AAVrh.69 WO2005033321 SEQ ID NO: 39
  • AAVrh.70 WO2005033321 SEQ ID NO: 20
  • AAVrh.72 WO2005033321 SEQ ID NO: 9
  • variants thereof including, but not limited to, AAVcy.2, AAVcy.3, AAVcy.4, AAVcy.5, AAVcy.6, AAVrh.12, AAVrh.17, AAVrh.18, AAVrh.19, AAVrh.21, AAVrh.22, AAVrh.23, AAVrh.24, AAVrh.25, AAVrh.25/42 15, AAVrh.31,
  • Non limiting examples of variants include SEQ ID NO: 13, 15, 17, 19, 24, 36, 40, 45, 47, 48, 51-54, 60-62, 64-77, 79, 80, 82, 89, 90, 93-95, 98, 100, 101, , 109- 113, 118-120, 124, 126, 131, 139, 142, 151, 154, 158, 161, 162, 165-183, 202, 204-212, 215, 219, 224-236, of WO2005033321, the contents of which are herein incorporated by reference in their entirety.
  • the AAV serotype may be, or have, a sequence as described in International Publication No. WO2015168666, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, AAVrh8R (SEQ ID NO: 9 of
  • the AAV serotype may be, or have, a sequence as described in United States Patent No. US9233131, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, AAVhEl .
  • the AAV serotype may be, or have, a sequence as described in United States Patent Publication No. US20150376607, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, AAV-PAEC (SEQ ID NO: 1 of
  • the AAV serotype may be, or have, a sequence as described in United States Patent No. US9163261, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, AAV-2-pre-miRNA-101 (SEQ ID NO: 1 US9163261), or variants thereof.
  • the AAV serotype may be, or have, a sequence as described in United States Patent Publication No. US20150376240, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, AAV-8h (SEQ ID NO: 6 of
  • US20150376240 AAV-8b (SEQ ID NO: 5 of US20150376240), AAV-h (SEQ ID NO: 2 of US20150376240), AAV-b (SEQ ID NO: 1 of US20150376240), or variants thereof.
  • the AAV serotype may be, or have, a sequence as described in United States Patent Publication No. US20160017295, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, AAV SM 10-2 (SEQ ID NO: 22 of US20160017295), AAV Shuffle 100-1 (SEQ ID NO: 23 of US20160017295), AAV Shuffle 100-3 (SEQ ID NO: 24 of US20160017295), AAV Shuffle 100-7 (SEQ ID NO: 25 of US20160017295), AAV Shuffle 10-2 (SEQ ID NO: 34 of US20160017295), AAV Shuffle 10-6 (SEQ ID NO: 35 of US20160017295), AAV Shuffle 10-8 (SEQ ID NO: 36 of US20160017295), AAV Shuffle 100-2 (SEQ ID NO: 37 of US20160017295), AAV SM 10-1 (SEQ ID NO: 38 of US20160017295), AAV SM 10-8 (SEQ ID NO:
  • the AAV serotype may be, or have, a sequence as described in United States Patent Publication No. US20150238550, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, BNP61 AAV (SEQ ID NO: 1 of
  • BNP62 AAV SEQ ID NO: 3 of US20150238550
  • BNP63 AAV SEQ ID NO: 4 of US20150238550
  • the AAV serotype may be or may have a sequence as described in United States Patent Publication No. US20150315612, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, AAVrh.50 (SEQ ID NO: 108 of
  • AAV54.4R/hu.27 (SEQ ID No: 64 of US20150315612), AAV46.2/hu.28 (SEQ ID No: 68 of US20150315612), AAV46.6/hu.29 (SEQ ID No: 69 of US20150315612), AAV128.1/hu.43 (SEQ ID No: 80 of US20150315612), or variants thereof.
  • the AAV serotype may be, or have, a sequence as described in International Publication No. WO2015121501, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, true type AAV (ttAAV) (SEQ ID NO: 2 of WO2015121501), "UPenn AAV10” (SEQ ID NO: 8 of WO2015121501), “Japanese AAV10” (SEQ ID NO: 9 of WO2015121501), or variants thereof.
  • true type AAV ttAAV
  • UPenn AAV10 SEQ ID NO: 8 of WO2015121501
  • Japanese AAV10 Japanese AAV10
  • AAV capsid serotype selection or use may be from a variety of species.
  • the AAV may be an avian AAV (AAAV).
  • the AAAV serotype may be, or have, a sequence as described in United States Patent No. US 9238800, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, AAAV (SEQ ID NO: 1, 2, 4, 6, 8, 10, 12, and 14 of US 9,238,800), or variants thereof.
  • the AAV may be a bovine AAV (BAAV).
  • BAAV serotype may be, or have, a sequence as described in United States Patent No. US 9,193,769, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, BAAV (SEQ ID NO: 1 and 6 of US 9193769), or variants thereof.
  • BAAV serotype may be or have a sequence as described in United States Patent No. US7427396, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, BAAV (SEQ ID NO: 5 and 6 of US7427396), or variants thereof.
  • the AAV may be a caprine AAV.
  • the caprine AAV serotype may be, or have, a sequence as described in United States Patent No. US7427396, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, caprine AAV (SEQ ID NO: 3 of US7427396), or variants thereof.
  • the AAV may be engineered as a hybrid AAV from two or more parental serotypes.
  • the AAV may be AAV2G9 which comprises sequences from AAV2 and AAV9.
  • the AAV2G9 AAV serotype may be, or have, a sequence as described in United States Patent Publication No. US20160017005, the contents of which are herein incorporated by reference in its entirety.
  • the AAV may be a serotype generated by the AAV9 capsid library with mutations in amino acids 390-627 (VP1 numbering) as described by Pulichla et al.
  • the serotype and corresponding nucleotide and amino acid substitutions may be, but is not limited to, AAV9.1 (G1594C; D532H), AAV6.2 (T1418A and T1436X; V473D and I479K), AAV9.3 (T1238A; F413Y), AAV9.4 (T1250C and A1617T; F417S), AAV9.5
  • the AAV serotype may be, or have, a sequence as described in International Publication No. WO2016049230, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to AAVF1/HSC1 (SEQ ID NO: 2 and 20 of WO2016049230), AAVF2/HSC2 (SEQ ID NO: 3 and 21 of WO2016049230), AAVF3/HSC3 (SEQ ID NO: 5 and 22 of WO2016049230), AAVF4/HSC4 (SEQ ID NO: 6 and 23 of WO2016049230), AAVF5/HSC5 (SEQ ID NO: 11 and 25 of WO2016049230), AAVF6/HSC6 (SEQ ID NO: 7 and 24 of WO2016049230), AAVF7/HSC7 (SEQ ID NO: 8 and 27 of WO2016049230), AAVF8/HSC8 (SEQ ID NO: 9 and 28 of WO20160490
  • WO2016049230 AAVF11/HSC11 (SEQ ID NO: 4 and 26 of WO2016049230), AAVF12/HSC12 (SEQ ID NO: 12 and 30 of WO2016049230), AAVF13/HSC13 (SEQ ID NO: 14 and 31 of
  • WO2016049230 AAVF14/HSC14 (SEQ ID NO: 15 and 32 of WO2016049230), AAVF15/HSC15 (SEQ ID NO: 16 and 33 of WO2016049230), AAVF16/HSC16 (SEQ ID NO: 17 and 34 of
  • WO2016049230 AAVF17/HSC17 (SEQ ID NO: 13 and 35 of WO2016049230), or variants or derivatives thereof.
  • the AAV serotype may be, or have, a sequence as described in United States Patent No. US 8734809, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, AAV CBr-El (SEQ ID NO: 13 and 87 of US8734809), AAV CBr-E2 (SEQ ID NO: 14 and 88 of US8734809), AAV CBr-E3 (SEQ ID NO: 15 and 89 of US8734809), AAV CBr-E4 (SEQ ID NO: 16 and 90 of US8734809), AAV CBr-E5 (SEQ ID NO: 17 and 91 of US8734809), AAV CBr-e5 (SEQ ID NO: 18 and 92 of US8734809), AAV CBr-E6 (SEQ ID NO: 19 and 93 of US8734809), AAV CBr-E7 (SEQ ID NO: 20 and 94 of US87
  • the AAV serotype may be, or have, a sequence as described in International Publication No. WO2016065001, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to AAV CHt-P2 (SEQ ID NO: 1 and 51 of WO2016065001), AAV CHt-P5 (SEQ ID NO: 2 and 52 of WO2016065001), AAV CHt-P9 (SEQ ID NO: 3 and 53 of WO2016065001), AAV CBr-7.1 (SEQ ID NO: 4 and 54 of WO2016065001), AAV CBr-7.2 (SEQ ID NO: 5 and 55 of WO2016065001), AAV CBr-7.3 (SEQ ID NO: 6 and 56 of WO2016065001), AAV CBr-7.4 (SEQ ID NO: 7 and 57 of WO2016065001), AAV CBr-7.5 (SEQ ID NO: 8 and 58 of WO2016065001),
  • WO2016065001 AAV CLv-L4 (SEQ ID NO: 15 and 65 of WO2016065001), AAV CLv-L5 (SEQ ID NO: 16 and 66 of WO2016065001), AAV CLv-L6 (SEQ ID NO: 17 and 67 of WO2016065001), AAV CLv-Kl (SEQ ID NO: 18 and 68 of WO2016065001), AAV CLv-K3 (SEQ ID NO: 19 and 69 of WO2016065001), AAV CLv-K6 (SEQ ID NO: 20 and 70 of WO2016065001), AAV CLv-Ml (SEQ ID NO: 21 and 71 of WO2016065001), AAV CLv-Ml 1 (SEQ ID NO: 22 and 72 of
  • WO2016065001 AAV CLv-M2 (SEQ ID NO: 23 and 73 of WO2016065001), AAV CLv-M5 (SEQ ID NO: 24 and 74 of WO2016065001), AAV CLv-M6 (SEQ ID NO: 25 and 75 of
  • WO2016065001 AAV CLv-M7 (SEQ ID NO: 26 and 76 of WO2016065001), AAV CLv-M8 (SEQ ID NO: 27 and 77 of WO2016065001), AAV CLv-M9 (SEQ ID NO: 28 and 78 of
  • WO2016065001 AAV CHt-Pl (SEQ ID NO: 29 and 79 of WO2016065001), AAV CHt-P6 (SEQ ID NO: 30 and 80 of WO2016065001), AAV CHt-P8 (SEQ ID NO: 31 and 81 of WO2016065001), AAV CHt-6.1 (SEQ ID NO: 32 and 82 of WO2016065001), AAV CHt-6.10 (SEQ ID NO: 33 and 83 of WO2016065001), AAV CHt-6.5 (SEQ ID NO: 34 and 84 of WO2016065001), AAV CHt-6.6 (SEQ ID NO: 35 and 85 of WO2016065001), AAV CHt-6.7 (SEQ ID NO: 36 and 86 of
  • WO2016065001 AAV CSp-8.4 (SEQ ID NO: 40 and 90 of WO2016065001), AAV CSp-8.5 (SEQ ID NO: 41 and 91 of WO2016065001), AAV CSp-8.6 (SEQ ID NO: 42 and 92 of WO2016065001), AAV CSp-8.7 (SEQ ID NO: 43 and 93 of WO2016065001), AAV CSp-8.8 (SEQ ID NO: 44 and 94 of WO2016065001), AAV CSp-8.9 (SEQ ID NO: 45 and 95 of WO2016065001), AAV CBr-B7.3 (SEQ ID NO: 46 and 96 of WO2016065001), AAV CBr-B7.4 (SEQ ID NO: 47 and 97 of
  • WO2016065001 AAV3B (SEQ ID NO: 48 and 98 of WO2016065001), AAV4 (SEQ ID NO: 49 and 99 of WO2016065001), AAV5 (SEQ ID NO: 50 and 100 of WO2016065001), or variants or derivatives thereof.
  • the AAV serotype may be, or have, a modification as described in United States Publication No. US 20160361439, the contents of which are herein incorporated by reference in their entirety, such as but not limited to, Y252F, Y272F, Y444F, Y500F, Y700F, Y704F, Y730F, Y275F, Y281F, Y508F, Y576F, Y612G, Y673F, and Y720F of the wild-type AAVl, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV10, AAV11, AAV 12, and hybrids thereof.
  • the AAV serotype may be, or have, a mutation as described in United States Patent No. US 9546112, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, at least two, but not all the F129L, D418E, K53 IE, L584F, V598A and H642N mutations in the sequence of AAV6 (SEQ ID NO:4 of US 9546112), AAVl (SEQ ID NO:6 of US 9546112), AAV2, AAV3, AAV4, AAV5, AAV7, AAV9, AAV10 or AAVl 1 or derivatives thereof.
  • the AAV serotype may be, or have, an AAV6 sequence comprising the K53 IE mutation (SEQ ID NO:5 of US 95461 12).
  • the AAV serotype may be, or have, a mutation in the AAVl sequence, as described in in United States Publication No. US 20130224836, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, at least one of the surface-exposed tyrosine residues, preferably, at positions 252, 273, 445, 701, 705 and 731 of AAVl (SEQ ID NO: 2 of US 20130224836) substituted with another amino acid, preferably with a phenylalanine residue.
  • the AAV serotype may be, or have, a mutation in the AAV9 sequence, such as, but not limited to, at least one of the surface-exposed tyrosine residues, preferably, at positions 252, 272, 444, 500, 700, 704 and 730 of AAV2 (SEQ ID NO: 4 of US 20130224836) substituted with another amino acid, preferably with a phenylalanine residue.
  • the tyrosine residue at position 446 of AAV9 (SEQ ID NO: 6 US 20130224836) is substituted with a phenylalanine residue.
  • the serotype may be AAV2 or a variant thereof, as described in International Publication No.
  • the amino acid sequence of AAV2 may comprise N587A, E548A, or N708A mutations. In one embodiment, the amino acid sequence of any AAV may comprise a V708K mutation.
  • the AAV may be a serotype selected from any of those found in Table 1.
  • the AAV may comprise a sequence, fragment or variant thereof, of the sequences in Table 1.
  • the AAV may be encoded by a sequence, fragment or variant as described in Table 1.
  • AAVcy.2 (AAV13.3) 145 US20030138772 SEQ ID NO 15
  • AAVcy.3 (AAV24.1) 147 US20030138772 SEQ ID NO 16
  • AAVcy.4 (AAV27.3) 149 US20030138772 SEQ ID NO 17
  • AAVcy.5 (AAV7.2) 152 US20030138772 SEQ ID NO 18
  • AAVcy.6 (AAV16.3) 154 US20030138772 SEQ ID NO 10
  • AAVhErl.23 (AAVhEr2.29) 172 US9233131 SEQ ID NO: 53
  • AAVhu. lO (AAV16.8) 186 US20150315612 SEQ ID NO: 56
  • AAVhu.10 (AAV16.8) 187 US20150315612 SEQ ID NO: 156
  • AAVhu. l l (AAV16.12) 188 US20150315612 SEQ ID NO: 57
  • AAVhu.15 (AAV33.4) 199 US20150315612 SEQ ID NO: 50
  • AAVhu.16 (AAV33.8) 202 US20150315612 SEQ ID NO: 51
  • AAVhu.17 (AAV33.12) 204 US20150315612 SEQ ID NO: 4
  • AAVhu.39 (AAVLG-9) 257 US20150315612 SEQ ID NO: 24
  • AAVhu.40 (AAV114.3) 261 US20150315612 SEQ ID No: 11
  • AAVhu.41 (AAV127.2) 263 US20150315612 SEQ ID NO: 6
  • AAVhu.42 (AAV127.5) 265 US20150315612 SEQ ID NO: 8
  • AAVhu.43 (AAV128.1) 268 US20150315612 SEQ ID NO: 80
  • AAVhu.44 (AAV128.3) 270 US20150315612 SEQ ID NO: 81
  • AAVhu.48 (AAV130.4) 283 US20150315612 SEQ ID NO: 78
  • AAVhu.53 (AAV145.1) 297 US20150315612 SEQ ID NO 176
  • AAVhu.54 (AAV145.5) 299 US20150315612 SEQ ID No: 177
  • AAVhu.56 (AAV145.6) 302 US20150315612 SEQ ID NO: 168
  • AAVhu.56 (AAV145.6) 303 US20150315612 SEQ ID NO: 192
  • AAVhu.6 (AAV3.1) 309 US20150315612 SEQ ID NO: 5
  • AAVhu.6 (AAV3.1) 310 US20150315612 SEQ ID NO: 84
  • AAVhu.60 (AAV161.10) 312 US20150315612 SEQ ID NO: 170
  • AAVhu.61 (AAV161.6) 314 US20150315612 SEQ ID NO: 174
  • AAVhu.7 (AAV7.3) 324 US20150315612 SEQ ID NO: 55
  • AAVhu.9 (AAV3.1) 329 US20150315612 SEQ ID NO: 58
  • AAVhu.9 (AAV3.1) 330 US20150315612 SEQ ID NO: 155
  • AAV-LK06 342 US20150376607 SEQ ID NO: 34
  • AAVrh.10 (AAV44.2) 396 US20030138772 SEQ ID NO: 81
  • AAVrh.12 (AAV42.1b) 398 US20030138772 SEQ ID NO: 30
  • AAVrh.14 (AAV42.3a) 404 US20030138772 SEQ ID NO: 32
  • AAVrh.17 (AAV42.5a) 406 US20030138772 SEQ ID NO: 34
  • AAVrh.18 (AAV42.5b) 408 US20030138772 SEQ ID NO: 29
  • AAVrh.19 (AAV42.6b) 410 US20030138772 SEQ ID NO: 38
  • AAVrh.21 (AAV42.10) 415 US20030138772 SEQ ID NO: 35
  • AAVrh.48 (AAV1-7) 460 US20150315612 SEQ ID NO: 32
  • AAVrh.49 (AAV1-8) 461 US20150315612 SEQ ID NO: 25
  • AAVrh.49 (AAV1-8) 462 US20150315612 SEQ ID NO: 103
  • AAVrh.50 (AAV2-4) 463 US20150315612 SEQ ID NO: 23
  • AAVrh.50 (AAV2-4) 464 US20150315612 SEQ ID NO: 108
  • AAVrh.51 (AAV2-5) 465 US20150315612 SEQ ID No: 22
  • AAVrh.51 (AAV2-5) 466 US20150315612 SEQ ID NO: 104
  • AAVrh.52 (AAV3-9) 467 US20150315612 SEQ ID NO: 18
  • AAVrh.52 (AAV3-9) 468 US20150315612 SEQ ID NO: 96
  • AAVrh.53 (AAV3-11) 470 US20150315612 SEQ ID NO: 17
  • AAVrh.53 (AAV3-11) 471 US20150315612 SEQ ID NO: 186
  • AAVrh.55 (AAV4-19) 475 US20150315612 SEQ ID NO: 117
  • AAVrh.61 (AAV2-3) 488 US20150315612 SEQ ID NO: 21
  • AAVrh.62 (AAV2-15) 489 US20150315612 SEQ ID No: 33
  • AAVrh.62 (AAV2-15) 490 US20150315612 SEQ ID NO: 114
  • BAAV bovine AAV 516 US9193769 SEQ ID NO: 8
  • BAAV bovine AAV 517 US9193769 SEQ ID NO: 10
  • BAAV bovine AAV 518 US9193769 SEQ ID NO: 4
  • BAAV bovine AAV 520 US9193769 SEQ ID NO: 6
  • BAAV bovine AAV 521 US9193769 SEQ ID NO: 1
  • BAAV bovine AAV 522 US9193769 SEQ ID NO: 5
  • BAAV bovine AAV 523 US9193769 SEQ ID NO: 3
  • BAAV bovine AAV 525 US7427396 SEQ ID NO: 5
  • BAAV bovine AAV 526 US7427396 SEQ ID NO: 6
  • AAAV (Avian AAV) 536 US9238800 SEQ ID NO: 12
  • AAAV (Avian AAV) 537 US9238800 SEQ ID NO: 2
  • AAAV (Avian AAV) 538 US9238800 SEQ ID NO: 6
  • AAAV (Avian AAV) 539 US9238800 SEQ ID NO: 4
  • AAAV (Avian AAV) 540 US9238800 SEQ ID NO: 8
  • AAAV (Avian AAV) 541 US9238800 SEQ ID NO: 14
  • AAAV (Avian AAV) 542 US9238800 SEQ ID NO: 10
  • AAAV (Avian AAV) 543 US9238800 SEQ ID NO: 15
  • AAAV (Avian AAV) 544 US9238800 SEQ ID NO: 5
  • AAAV (Avian AAV) 545 US9238800 SEQ ID NO: 9
  • AAAV (Avian AAV) 546 US9238800 SEQ ID NO: 3
  • AAAV (Avian AAV) 547 US9238800 SEQ ID NO: 7
  • AAAV (Avian AAV) 548 US9238800 SEQ ID NO: 11
  • AAAV (Avian AAV) 549 US9238800 SEQ ID NO: 13
  • AAAV (Avian AAV) 550 US9238800 SEQ ID NO: 1
  • AAV3B 870 WO2016065001 SEQ ID NO: 98
  • rh74 (RHM4-1) 1017 US2015023924A1 SEQ ID NO: 11 rh74 (RHM15-1) 1018 US2015023924A1 SEQ ID NO 12 rh74 (RHM15-2) 1019 US2015023924A1 SEQ ID NO 13 rh74 (RHM15-3/RHM15-5) 1020 US2015023924A1 SEQ ID NO 14 rh74 (RHM15-4) 1021 US2015023924A1 SEQ ID NO 15 rh74 (RHM15-6) 1022 US2015023924A1 SEQ ID NO 16
  • AAV2 (comprising lung
  • AAV2 (comprising lung

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PE2019002526A PE20200494A1 (es) 2017-06-15 2018-06-14 Polinucleotidos de aadc para el tratamiento de la enfermedad de parkinson
CA3065471A CA3065471A1 (en) 2017-06-15 2018-06-14 Aadc polynucleotides for the treatment of parkinson's disease
BR112019025122-7A BR112019025122A2 (pt) 2017-06-15 2018-06-14 polinucleotídeos de aadc para o tratamento de mal de parkinson
EP18816860.3A EP3638315A4 (en) 2017-06-15 2018-06-14 AADC POLYNUCLEOTIDES FOR THE TREATMENT OF PARKINSON'S DISEASE
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EA201992604A EA201992604A1 (ru) 2017-09-05 2018-06-14 Полинуклеотиды aadc для лечения болезни паркинсона
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CONC2019/0013707A CO2019013707A2 (es) 2017-06-15 2019-12-05 Polinucleótidos de aadc para el tratamiento de la enfermedad de parkinson
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Cited By (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10414803B2 (en) 2015-05-11 2019-09-17 Ucl Business Plc Capsid
WO2019222328A1 (en) * 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
WO2019241486A1 (en) 2018-06-13 2019-12-19 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
WO2020023612A1 (en) 2018-07-24 2020-01-30 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
WO2020072849A1 (en) 2018-10-04 2020-04-09 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
WO2020072844A1 (en) 2018-10-05 2020-04-09 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding aav production proteins
WO2020081490A1 (en) 2018-10-15 2020-04-23 Voyager Therapeutics, Inc. EXPRESSION VECTORS FOR LARGE-SCALE PRODUCTION OF rAAV IN THE BACULOVIRUS/Sf9 SYSTEM
WO2020223274A1 (en) 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS
WO2021007382A1 (en) * 2019-07-09 2021-01-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
WO2021030125A1 (en) 2019-08-09 2021-02-18 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
WO2021041485A1 (en) 2019-08-26 2021-03-04 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2021119615A1 (en) * 2019-12-12 2021-06-17 Meiragtx Uk Ii Limited Methods for treating parkinson's disease
WO2022032153A1 (en) 2020-08-06 2022-02-10 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
WO2022187473A2 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022187548A1 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
US11525139B2 (en) 2016-08-23 2022-12-13 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
WO2023093905A1 (zh) 2021-11-29 2023-06-01 上海瑞宏迪医药有限公司 Aadc、gdnf多核苷酸及其用于治疗帕金森病
WO2023155918A1 (zh) 2022-02-21 2023-08-24 上海瑞宏迪医药有限公司 Vegf结合分子及其医药用途
US11759506B2 (en) 2017-06-15 2023-09-19 Voyager Therapeutics, Inc. AADC polynucleotides for the treatment of Parkinson's disease
EP3943503A4 (en) * 2019-01-30 2023-10-18 Shanghaitech University Adeno-associated virus having variant capsid protein and use thereof
US11807867B2 (en) 2020-02-21 2023-11-07 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
US11975056B2 (en) 2014-11-05 2024-05-07 Voyager Therapeutics, Inc. AADC polynucleotides for the treatment of Parkinson's disease
US12116385B2 (en) 2021-10-08 2024-10-15 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
WO2025040166A1 (zh) 2023-08-23 2025-02-27 上海瑞宏迪医药有限公司 药物组合物及其用途
WO2025051805A1 (en) * 2023-09-04 2025-03-13 Uniqure Biopharma B.V. Novel neurotropic recombinant adeno-associated virus particles

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10898585B2 (en) 2017-04-14 2021-01-26 Ptc Therapeutics .Inc. Gene therapy for AADC deficiency
EP3727468A4 (en) 2017-12-19 2021-09-22 Akouos, Inc. AAV-MEDIATED THERAPEUTIC ANTIBODIES DELIVERY TO THE INTERNAL EAR
US20230075314A1 (en) * 2019-04-29 2023-03-09 Voyager Therapeutics, Inc. VECTORIZED ANTIBODIES (vAb) AND USES THEREOF
SG11202111375RA (en) 2019-04-29 2021-11-29 Univ Pennsylvania Novel aav capsids and compositions containing same
CN115135347A (zh) * 2019-12-12 2022-09-30 梅里特斯英国第二有限公司 治疗帕金森病的方法
CA3177869A1 (en) * 2020-05-05 2021-11-11 Duke University Cross-species compatible adeno-associated virus compositions and methods of use thereof
CA3190059A1 (en) * 2020-08-17 2022-02-24 Yu TAO Nucleic acid construct for increasing adeno-associated virus yield, and construction method therefor
IL303317A (en) 2020-12-01 2023-07-01 Akouos Inc ANTI-NATURAL ANTIBODY STRUCTURES AND RELATED METHODS FOR THE TREATMENT OF SYMPTOMS ASSOCIATED WITH VESTIBULAR SWANNOMA
CA3203975A1 (en) 2020-12-03 2022-06-09 Battelle Memorial Institute Polymer nanoparticle and dna nanostructure compositions and methods for non-viral delivery
CA3216359A1 (en) 2021-04-07 2022-10-13 Battelle Memorial Institute Rapid design, build, test, and learn technologies for identifying and using non-viral carriers
AU2024353375A1 (en) 2023-09-29 2026-04-09 Battelle Memorial Institute Polymer nanoparticle compositions for in vivo expression of polypeptides
WO2025122954A1 (en) 2023-12-08 2025-06-12 Battelle Memorial Institute Use of dna origami nanostructures for molecular information based data storage systems

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016073693A2 (en) * 2014-11-05 2016-05-12 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease

Family Cites Families (455)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR2640638B1 (fr) 1988-12-20 1991-02-15 Commissariat Energie Atomique Bioreacteur et dispositif pour la culture de cellules animales
WO1991018088A1 (en) 1990-05-23 1991-11-28 The United States Of America, Represented By The Secretary, United States Department Of Commerce Adeno-associated virus (aav)-based eucaryotic vectors
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
GB9027917D0 (en) 1990-12-21 1991-02-13 Ici Plc Expression systems
US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
US5693531A (en) 1993-11-24 1997-12-02 The United States Of America As Represented By The Department Of Health And Human Services Vector systems for the generation of adeno-associated virus particles
ATE386131T1 (de) 1994-04-13 2008-03-15 Univ Rockefeller Aav-vermittelte überbringung von dna in zellen des nervensystems
US20020159979A1 (en) 1994-06-06 2002-10-31 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
KR100364470B1 (ko) 1994-07-15 2003-02-05 이데미쓰세끼유가가꾸가부시끼가이샤 고강성 폴리프로필렌계수지 및 그것을 이용한 블로우성형체
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
US5652224A (en) 1995-02-24 1997-07-29 The Trustees Of The University Of Pennsylvania Methods and compositions for gene therapy for the treatment of defects in lipoprotein metabolism
US5741657A (en) 1995-03-20 1998-04-21 The Regents Of The University Of California Fluorogenic substrates for β-lactamase and methods of use
US6281010B1 (en) 1995-06-05 2001-08-28 The Trustees Of The University Of Pennsylvania Adenovirus gene therapy vehicle and cell line
US5756283A (en) 1995-06-05 1998-05-26 The Trustees Of The University Of Pennsylvania Method for improved production of recombinant adeno-associated viruses for gene therapy
US5688676A (en) 1995-06-07 1997-11-18 Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US6676935B2 (en) 1995-06-27 2004-01-13 Cell Genesys, Inc. Tissue specific adenoviral vectors
US6197293B1 (en) 1997-03-03 2001-03-06 Calydon, Inc. Adenovirus vectors specific for cells expressing androgen receptor and methods of use thereof
WO1997008298A1 (en) 1995-08-30 1997-03-06 Genzyme Corporation Chromatographic purification of adenovirus and aav
US6265389B1 (en) 1995-08-31 2001-07-24 Alkermes Controlled Therapeutics, Inc. Microencapsulation and sustained release of oligonucleotides
US5846528A (en) 1996-01-18 1998-12-08 Avigen, Inc. Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence
US5962313A (en) 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
US5858351A (en) 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
US5952221A (en) 1996-03-06 1999-09-14 Avigen, Inc. Adeno-associated virus vectors comprising a first and second nucleic acid sequence
US7026468B2 (en) 1996-07-19 2006-04-11 Valentis, Inc. Process and equipment for plasmid purification
US20020037867A1 (en) 1999-02-26 2002-03-28 James M. Wilson Method for recombinant adeno-associated virus-directed gene therapy
EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
AU722375B2 (en) 1996-09-06 2000-08-03 Trustees Of The University Of Pennsylvania, The Methods using cre-lox for production of recombinant adeno-associated viruses
WO1998010087A1 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania Chimpanzee adenovirus vectors
JP2001500497A (ja) 1996-09-06 2001-01-16 トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組換えアデノ随伴ウイルスに指図される遺伝子治療の方法
US5866552A (en) 1996-09-06 1999-02-02 The Trustees Of The University Of Pennsylvania Method for expressing a gene in the absence of an immune response
WO1998011244A2 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secretary, Department Of Health And Human Services Aav4 vector and uses thereof
WO1998022588A2 (en) 1996-11-20 1998-05-28 Introgen Therapeutics, Inc. An improved method for the production and purification of adenoviral vectors
US7732129B1 (en) 1998-12-01 2010-06-08 Crucell Holland B.V. Method for the production and purification of adenoviral vectors
JP2001506133A (ja) 1996-12-18 2001-05-15 ターゲティッド ジェネティクス コーポレイション 組換えａａｖベクターの産生における使用のための、ａａｖスプリット−パッケージング遺伝子およびこのような遺伝子を含む細胞株
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6710036B2 (en) 1997-07-25 2004-03-23 Avigen, Inc. Induction of immune response to antigens expressed by recombinant adeno-associated virus
US6251677B1 (en) 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
CA2304131A1 (en) 1997-09-19 1999-04-01 James M. Wilson Method for gene transfer using bcl2 and compositions useful therein
WO1999014354A1 (en) 1997-09-19 1999-03-25 The Trustees Of The University Of The Pennsylvania Methods and vector constructs useful for production of recombinant aav
EP1015619A1 (en) 1997-09-19 2000-07-05 The Trustees Of The University Of Pennsylvania Methods and cell line useful for production of recombinant adeno-associated viruses
US6642051B1 (en) 1997-10-21 2003-11-04 Targeted Genetics Corporation Amplifiable adeno-associated virus(AAV) packaging cassettes for the production of recombinant AAV vectors
IT1297074B1 (it) 1997-11-21 1999-08-03 Angeletti P Ist Richerche Bio Forme ormone-dipendenti delle proteine rep del virus adeno-associato (aav-2), sequenze di dna codificanti per esse, vettori che le
NZ505325A (en) 1997-12-23 2003-07-25 Crucell Holland B Adeno-associated virus and adenovirus chimeric recombinant viruses useful for the integration of foreign genetic information into the chromosomal dna of target cells
US6410300B1 (en) 1998-01-12 2002-06-25 The University Of North Carolina At Chapel Hill Methods and formulations for mediating adeno-associated virus (AAV) attachment and infection and methods for purifying AAV
WO1999043360A1 (en) 1998-02-26 1999-09-02 The Trustees Of The University Of Pennsylvania Stable protection from dystrophic sarcolemmal degeneration and restoration of the sarcoglycan complex
US6953690B1 (en) 1998-03-20 2005-10-11 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US6521426B1 (en) 1998-04-08 2003-02-18 Istituto Di Ricerche Di Biologia Molecolare P. Angeletti S.P.A. Preparation of recombinant adenovirus carrying a rep gene of adeno-associated virus
FR2778413B1 (fr) 1998-05-07 2000-08-04 Immunotech Sa Nouveaux reactifs et methode de lyse des erythrocytes
EP1078096A1 (en) 1998-05-11 2001-02-28 Ariad Gene Therapeutics, Inc. Multiviral compositions and uses thereof
EP1849872A1 (en) 1998-05-20 2007-10-31 University Of Iowa Research Foundation Adeno-associated virus vectors and uses thereof
US6436392B1 (en) 1998-05-20 2002-08-20 University Of Iowa Research Foundation Adeno-associated virus vectors
EP1621626B1 (en) 1998-05-27 2009-07-08 Genzyme Corporation AAV Vectors for the manufacture of medicaments for convection enhanced delivery
WO1999061066A2 (en) 1998-05-27 1999-12-02 Avigen, Inc. Convection-enhanced delivery of aav vectors
US6146874A (en) 1998-05-27 2000-11-14 University Of Florida Method of preparing recombinant adeno-associated virus compositions
AU4185699A (en) 1998-05-27 1999-12-13 Avigen, Inc. Adeno-associated viral vector-mediated expression of factor viii activity
WO1999061601A2 (en) 1998-05-28 1999-12-02 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav5 vector and uses thereof
US6984517B1 (en) 1998-05-28 2006-01-10 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector and uses thereof
GB2338236B (en) 1998-06-13 2003-04-09 Aea Technology Plc Microbiological cell processing
US6900049B2 (en) 1998-09-10 2005-05-31 Cell Genesys, Inc. Adenovirus vectors containing cell status-specific response elements and methods of use thereof
WO2000022152A1 (en) 1998-10-13 2000-04-20 Avigen, Inc. Compositions and methods for producing recombinant adeno-associated virus
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6221349B1 (en) * 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
EP1124976A1 (en) 1998-10-27 2001-08-22 Crucell Holland B.V. Improved aav vector production
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
US6689600B1 (en) 1998-11-16 2004-02-10 Introgen Therapeutics, Inc. Formulation of adenovirus for gene therapy
US6759050B1 (en) 1998-12-03 2004-07-06 Avigen, Inc. Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith
US6225113B1 (en) 1998-12-04 2001-05-01 Genvec, Inc. Use of trans-activation and cis-activation to modulate the persistence of expression of a transgene in an at least E4-deficient adenovirus
US6387368B1 (en) 1999-02-08 2002-05-14 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
DE19905501B4 (de) 1999-02-10 2005-05-19 MediGene AG, Gesellschaft für molekularbiologische Kardiologie und Onkologie Verfahren zur Herstellung eines rekombinanten Adeno-assoziierten Virus, geeignete Mittel hierzu sowie Verwendung zur Herstellung eines Arzneimittels
US6509150B1 (en) 1999-03-05 2003-01-21 Universite De Nantes Compositions and methods for recombinant Adeno-Associated Virus production
JP4693244B2 (ja) 1999-03-18 2011-06-01 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
EP1183389A2 (en) 1999-04-30 2002-03-06 University of Florida Adeno-associated virus-delivered ribozyme compositions and methods of use
CA2375098A1 (en) 1999-06-02 2000-12-14 Trustees Of The University Of Pennsylvania Compositions and methods useful for production of recombinant viruses which require helper viruses
CN1378593A (zh) 1999-08-20 2002-11-06 约翰斯霍普金斯大学医学院 构建融合文库的方法和组合物及该文库的用途
US6365394B1 (en) 1999-09-29 2002-04-02 The Trustees Of The University Of Pennsylvania Cell lines and constructs useful in production of E1-deleted adenoviruses in absence of replication competent adenovirus
JP2003523320A (ja) 1999-09-29 2003-08-05 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ウイルスベクターの迅速なｐｅｇ改変方法、高められた遺伝子形質導入のための組成物、高められた物理的安定性を伴う組成物、およびそのための用途
US7241447B1 (en) 1999-10-07 2007-07-10 University Of Iowa Research Foundation Adeno-associated virus vectors and uses thereof
EP1224313A1 (en) 1999-10-07 2002-07-24 University of Iowa Research Foundation Adeno-associated viruses and uses thereof
WO2001032711A2 (en) 1999-10-21 2001-05-10 Board Of Trustees Of The University Of Arkansas Adeno-associated virus aav rep78 major regulatory protein, mutants thereof and uses thereof
AU3642601A (en) 1999-11-05 2001-05-30 Avigen, Inc. Ecdysone-inducible adeno-associated virus expression vectors
US6582692B1 (en) 1999-11-17 2003-06-24 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
CA2392299A1 (en) 1999-12-10 2001-06-14 Victor Rivera Methods for expression of genes in primates
US7638120B2 (en) 2000-03-14 2009-12-29 Thomas Jefferson University High transgene expression of a pseudotyped adeno-associated virus type
CA2373110A1 (en) 2000-03-14 2001-09-20 Neurologix, Inc. Production of chimeric capsid vectors
US6468524B1 (en) 2000-03-22 2002-10-22 The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services AAV4 vector and uses thereof
US6855314B1 (en) 2000-03-22 2005-02-15 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector for transducing brain cells and lung cells
US7048920B2 (en) 2000-03-24 2006-05-23 Cell Genesys, Inc. Recombinant oncolytic adenovirus for human melanoma
GB0009887D0 (en) 2000-04-20 2000-06-07 Btg Int Ltd Cytotoxic agents
US20030013189A1 (en) 2000-04-28 2003-01-16 Wilson James M. Compositions and methods useful for non-invasive delivery of therapeutic molecules to the bloodstream
AU2001257611A1 (en) 2000-04-28 2001-11-12 Avigen, Inc. Polynucleotides for use in recombinant adeno-associated virus virion production
US7056502B2 (en) 2000-04-28 2006-06-06 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids
CA2409674C (en) 2000-05-23 2010-05-11 Neurologix, Inc. Glutamic acid decarboxylase (gad) based delivery systems
WO2001096587A2 (en) 2000-06-13 2001-12-20 The Children's Hospital Of Philadelphia Methods for administering recombinant adeno-associated virus virions to humans previously exposed to adeno-associated virus
EP1302542B1 (en) 2000-07-18 2007-06-13 Takeda Pharmaceutical Company Limited Novel physiologically active peptide and use thereof
US6329181B1 (en) 2000-08-07 2001-12-11 Neurologix, Inc. Helper functions for recombinant vector production
US6593123B1 (en) 2000-08-07 2003-07-15 Avigen, Inc. Large-scale recombinant adeno-associated virus (rAAV) production and purification
DE60129229T2 (de) 2000-08-17 2008-05-08 Ozawa, Keiya Adeno-assoziierte virus-vermittelte übertragung von angiogenesefaktoren
DE10044384A1 (de) 2000-09-08 2002-04-18 Medigene Ag Wirtszellen zur Verpackung von rekombinantem Adeno-assoziiertem Virus (rAAV), Verfahren zu ihrer Herstellung und deren Verwendung
FR2813891B1 (fr) 2000-09-14 2005-01-14 Immunotech Sa Reactif multifonctionnel pour erythrocytes mettant en jeu des carbamates et applications
GB0024550D0 (https=) 2000-10-06 2000-11-22 Oxford Biomedica Ltd
JP2002153278A (ja) 2000-11-22 2002-05-28 Hisamitsu Pharmaceut Co Inc ウイルスベクターの製造に用いられる細胞、その製法およびその細胞を用いたウイルスベクターの製造方法
WO2002070719A2 (en) 2001-01-19 2002-09-12 Trustees Of The University Of Pennsylvania Regulatable gene expression system
US7588757B2 (en) 2001-03-14 2009-09-15 Genzyme Corporation Methods of treating Parkinson's disease using recombinant adeno-associated virus virions
JP2004532822A (ja) 2001-03-14 2004-10-28 アビジェン， インコーポレイテッド ビリオンの管逆行感染による組換えアデノ随伴ウイルス媒介遺伝子移入
US6841357B1 (en) 2001-03-30 2005-01-11 Stratagene California Method and kits for titering adeno-associated virus vectors
US7182944B2 (en) 2001-04-25 2007-02-27 The United States Of America As Represented By The Department Of Health And Human Services Methods of increasing distribution of nucleic acids
US20030096264A1 (en) 2001-06-18 2003-05-22 Psychiatric Genomics, Inc. Multi-parameter high throughput screening assays (MPHTS)
EP1409748B1 (en) 2001-06-22 2011-10-26 The Trustees of The University of Pennsylvania Recombinant Adenoviruses comprising simian adenovirus proteins and uses thereof.
US20040136963A1 (en) 2001-06-22 2004-07-15 The Trustees Of The University Of Pennsylvania Simian adenovirus vectors and methods of use
US8241622B2 (en) 2001-07-13 2012-08-14 University Of Iowa Research Foundation Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences
EP1279740A1 (en) 2001-07-26 2003-01-29 Vrije Universiteit Brussel Recombinant vector derived from adeno-associated virus for gene therapy
JP2004538005A (ja) 2001-08-08 2004-12-24 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア シアル酸に結合するタンパク質を有するウイルスベクターの精製法
US20030092161A1 (en) 2001-09-19 2003-05-15 The Trustees Of The University Of Pennsylvania Compositions and methods for production of recombinant viruses, and uses therefor
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
WO2003042361A2 (en) 2001-11-09 2003-05-22 Government Of The United States Of America, Department Of Health And Human Services Production of adeno-associated virus in insect cells
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
AU2002365366B2 (en) 2001-11-21 2007-05-10 The Trustees Of The University Of Pennsylvania Simian adenovirus nucleic acid and amino acid sequences, vectors containing same, and methods of use
EP1944043A1 (en) 2001-11-21 2008-07-16 The Trustees of the University of Pennsylvania Simian adenovirus nucleic acid and amino acid sequences, vectors containing same, and methods of use
CA2469623C (en) 2001-12-12 2012-05-29 F H Faulding & Co Limited Composition for the preservation of viruses
DK2573170T3 (en) 2001-12-17 2018-04-09 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 9, vectors containing them, and their use
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
EP1463530A4 (en) 2001-12-19 2006-09-06 Lijun Wang ADENO-ASSOCIATED VIRUS-RELATED DELIVERY OF GDNF TO SKELETAL MUSCLES
CN1617938A (zh) 2002-01-16 2005-05-18 戴诺生物技术有限公司 从单个样品中分离核酸和蛋白质的方法
SI1504108T1 (sl) 2002-02-01 2013-07-31 Oxford Biomedica (Uk) Limited Lentiviralni vektor
GB0208390D0 (en) 2002-04-11 2002-05-22 Univ London Adeno-associated virus producer system
US20030198620A1 (en) 2002-04-16 2003-10-23 Keiya Ozawa Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions
ATE348153T1 (de) 2002-04-29 2007-01-15 Univ Pennsylvania Methode für die direkte gewinnung und amplifikation von integrierten viren aus zellulärer gewebe-dna
DE60327069D1 (de) 2002-04-30 2009-05-20 Oncolytics Biotech Inc Verbesserte reinigungsmethode für viren
SI1496944T1 (sl) 2002-05-01 2009-02-28 Univ Florida Izboljšani sistemi ekspresije RAAV za genetsko modifikacijo specifičnih kapsidnih proteinov
ATE360683T1 (de) 2002-05-14 2007-05-15 Merck & Co Inc Verfahren zur reinigung von adenovirus
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
WO2004020605A2 (en) 2002-08-29 2004-03-11 The Board Of Trustees Of The Leland Stanford Junior University Circular nucleic acid vectors, and methods for making and using the same
TW200509968A (en) 2002-11-01 2005-03-16 Elan Pharm Inc Prevention and treatment of synucleinopathic disease
EP1418185A1 (en) 2002-11-11 2004-05-12 Aventis Pharma Deutschland GmbH Use of EDG2 receptor in an animal model of heart failure
US7169612B2 (en) 2002-11-11 2007-01-30 Sanofi-Aventis Deutschland Gmbh Use of EDG2 receptor in an animal model of heart failure
US7510872B2 (en) 2003-02-26 2009-03-31 Nationwide Children's Hospital Recombinant adeno-associated virus production
US20070172460A1 (en) 2003-03-19 2007-07-26 Jurgen Kleinschmidt Random peptide library displayed on aav vectors
WO2004108922A2 (en) 2003-04-25 2004-12-16 The Trustees Of The University Of Pennsylvania Use of aav comprising a capsid protein from aav7 or aav8 for the delivery of genes encoding apoprotein a or e genes to the liver
US8927269B2 (en) 2003-05-19 2015-01-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Avian adenoassociated virus and uses thereof
US7261544B2 (en) 2003-05-21 2007-08-28 Genzyme Corporation Methods for producing preparations of recombinant AAV virions substantially free of empty capsids
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
JP4888876B2 (ja) 2003-06-13 2012-02-29 田平 武 アルツハイマー病の治療のための組換えアデノ随伴ウィルスベクター
SI1633772T1 (sl) 2003-06-19 2016-06-30 Genzyme Corporation AAV virioni z zmanjšano imunoreaktivnostjo in uporaba za ta namen
US7291498B2 (en) 2003-06-20 2007-11-06 The Trustees Of The University Of Pennsylvania Methods of generating chimeric adenoviruses and uses for such chimeric adenoviruses
CA2528511C (en) 2003-06-20 2015-11-03 The Trustees Of The University Of Pennsylvania Methods of generating chimeric adenoviruses and uses for such chimeric adenoviruses
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
WO2005012537A2 (en) 2003-07-25 2005-02-10 Genvec, Inc. Adenoviral vector-based vaccines
US7683036B2 (en) 2003-07-31 2010-03-23 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
WO2005030292A2 (en) 2003-09-26 2005-04-07 The Trustees Of The University Of Pennsylvania Methods, compositions and apparatus for delivering heterologous molecules to cells
EP2345731B1 (en) 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
ES2361000T3 (es) 2004-04-28 2011-06-13 The Trustees Of The University Of Pennsylvania Suministro secuencial de moléculas inmunogénicas mediante administraciones de un adenovirus y de un virus adeno-asociado.
WO2005116224A2 (en) 2004-05-18 2005-12-08 Children's Memorial Hospital Tetracycline-regulated adeno-associated viral (aav) vectors for gene delivery to the nervous system
BRPI0511764B8 (pt) 2004-06-01 2021-05-25 Avigen Inc método de prevenção de agregação de vírions de vírus adeno-associado recombinante (raav) em uma preparação purificada de virions raav
MX2007003850A (es) 2004-10-05 2007-11-21 Genzyme Corp Canula escalonada.
US7901921B2 (en) 2004-10-22 2011-03-08 Oncolytics Biotech Inc. Viral purification methods
CN1286981C (zh) 2004-11-30 2006-11-29 华中科技大学同济医学院附属同济医院 表达人类cyp2j2反义基因的重组腺相关病毒及其制备方法
CN101213305A (zh) 2004-12-09 2008-07-02 建新公司 转基因在哺乳动物中枢神经系统中的可调控型表达
US8614101B2 (en) 2008-05-20 2013-12-24 Rapid Pathogen Screening, Inc. In situ lysis of cells in lateral flow immunoassays
US7625570B1 (en) 2005-03-10 2009-12-01 The Regents Of The University Of California Methods for purifying adeno-associated virus
WO2006102072A2 (en) 2005-03-23 2006-09-28 The Trustees Of The University Of Pennsylvania Use of a pa131 polypeptide in treatment of atherosclerosis
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
WO2006119432A2 (en) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
JP5829372B2 (ja) 2005-05-02 2015-12-09 ジェンザイム・コーポレーション 神経代謝性疾患のための遺伝子治療
PL2578685T3 (pl) 2005-08-23 2020-01-31 The Trustees Of The University Of Pennsylvania Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania
EP1928557B1 (en) 2005-08-23 2018-06-06 The Regents of The University of California Reflux resistant cannula and system for chronic delivery of therapeutic agents using convection-enhanced delivery
EP1857552A1 (en) 2006-05-20 2007-11-21 Cargill Incorporated Thermostable xylose isomerase enzyme
EP2311966A3 (en) 2005-10-20 2012-09-05 Amsterdam Molecular Therapeutics (AMT) B.V. Improved AAV vectors produced in insect cells
WO2008016391A2 (en) 2006-01-31 2008-02-07 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
DK2019683T4 (da) 2006-04-25 2022-08-29 Univ California Indgivelse af vækstfaktorer til behandling af CNS-lidelser
EP2018421B1 (en) 2006-04-28 2012-12-19 The Trustees of the University of Pennsylvania Scalable production method for aav
EP2012822B1 (en) 2006-04-28 2010-01-20 The Trustees of the University of Pennsylvania Modified adenovirus hexon protein and uses thereof
WO2008027084A2 (en) 2006-04-28 2008-03-06 The Trustees Of The University Of Pennsylvania Modified aav vectors having reduced capsid immunogenicity and use thereof
US20080003565A1 (en) 2006-05-02 2008-01-03 Government Of The Us, As Represented By The Secretary, Department Of Health And Human Services Viral nucleic acid microarray and method of use
ES2465996T3 (es) 2006-05-25 2014-06-09 Sangamo Biosciences, Inc. Métodos y composiciones para la inactivación genética
US8486635B2 (en) 2006-05-30 2013-07-16 Mayo Foundation For Medical Education And Research Detecting and treating dementia
HUE031156T2 (en) 2006-06-07 2017-06-28 Genzyme Corp Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
ES2535877T3 (es) 2006-06-19 2015-05-18 Asklepios Biopharmaceutical, Inc. Genes de Factor VIII y Factor IX modificados y vectores para la terapia génica
CN103849629B (zh) 2006-06-21 2017-06-09 尤尼克尔Ip股份有限公司 具有经修饰的用于在昆虫细胞中产生aav的aav‑rep78翻译起始密码子的载体
US8329162B2 (en) 2006-07-21 2012-12-11 California Institute Of Technology Targeted gene delivery for dendritic cell vaccination
WO2008024998A2 (en) 2006-08-24 2008-02-28 Virovek, Inc. Expression in insect cells of genes with overlapping open reading frames, methods and compositions therefor
EP2097095B1 (en) 2006-11-29 2016-01-27 Nationwide Children's Hospital Myostatin inhibition for enhancing muscle and/or improving muscle function
SG178744A1 (en) 2007-02-02 2012-03-29 Biogen Idec Inc Use of semaphorin 6a for promoting myelination and oligodendrocyte differentiation
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
ES2714007T3 (es) 2007-04-09 2019-05-24 Univ Florida Composiciones de vectores rAAV que tienen proteínas de la cápside modificadas en tirosina y métodos para su uso
US9611302B2 (en) 2007-04-09 2017-04-04 University Of Florida Research Foundation, Inc. High-transduction-efficiency RAAV vectors, compositions, and methods of use
WO2008128251A1 (en) 2007-04-17 2008-10-23 The Children's Hospital Of Philadelphia Humanized viral vectors and methods of use thereof
EP2012122A1 (en) 2007-07-06 2009-01-07 Medigene AG Mutated parvovirus structural proteins as vaccines
ES2602610T3 (es) 2007-05-31 2017-02-21 Medigene Ag Proteína estructural mutada de un parvovirus
ES2635726T3 (es) 2007-06-06 2017-10-04 Genzyme Corporation Terapia génica para enfermedades de almacenamiento lisosómico
MX2009013120A (es) 2007-06-29 2010-01-15 Hoffmann La Roche Promotor.
EP2176283B1 (en) 2007-07-14 2016-11-02 University of Iowa Research Foundation Methods and compositions for treating brain diseases
DK2173888T3 (en) 2007-07-26 2016-11-28 Uniqure Ip Bv Baculovirus vectors comprising repeating coding sequences WITH differential preferred codons
US8420372B2 (en) 2007-09-04 2013-04-16 Alexander Bello Porcine adeno-associated viruses
ES2426091T3 (es) 2007-09-19 2013-10-21 Uniqure Ip B.V. Uso de maquinaria de replicación de AAV para producción de proteína mejorada
WO2009051421A2 (en) 2007-10-18 2009-04-23 Lg Electronics Inc. Method and system for transmitting and receiving signals
US8642341B2 (en) 2007-11-02 2014-02-04 Cornell University Materials and methods for gene mediated therapy of psychiatric disorders
CN102131920B (zh) 2007-11-28 2013-11-06 宾夕法尼亚大学托管会 猿猴亚家族C腺病毒SAdV-40、-31和-34及其应用
CN101883858B (zh) 2007-11-28 2015-07-22 宾夕法尼亚大学托管会 猿猴亚家族E腺病毒SAdV-39、-25.2、-26、-30、-37和-38及其应用
AU2008331905B2 (en) 2007-11-28 2014-09-18 The Trustees Of The University Of Pennsylvania Simian subfamily B adenovirus SAdV-28 and uses thereof
CN101977934B (zh) 2008-01-18 2014-09-17 健泰科生物技术公司 用于靶向k63连接的多聚遍在蛋白的方法和组合物
ES2751999T3 (es) 2008-01-29 2020-04-02 Applied Genetic Tech Corporation Producción recombinante de virus adeno-asociados usando células BHK en suspensión
JP2011512156A (ja) 2008-02-19 2011-04-21 アムステルダム モレキュラー セラピューティクス ビー．ブイ． 昆虫細胞におけるパルボウイルスｒｅｐ及びｃａｐタンパク質の発現の最適化
JP5661476B2 (ja) 2008-03-04 2015-01-28 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア サルアデノウイルスＳＡｄＶ−３６、−４２．１、−４２．２および−４４ならびにそれらの用途
US9265843B2 (en) 2008-03-27 2016-02-23 The Ohio State University Treatment of metabolic-related disorders using hypothalamic gene transfer of BDNF and compositions therefor
WO2009137006A2 (en) 2008-04-30 2009-11-12 The University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
WO2009134681A2 (en) 2008-04-30 2009-11-05 The Trustees Of The University Of Pennsylvania Aav7 viral vectors for targeted delivery of rpe cells
WO2010011404A2 (en) 2008-05-20 2010-01-28 Eos Neuroscience, Inc. Vectors for delivery of light-sensitive proteins and methods of use
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
WO2009154452A1 (en) 2008-06-17 2009-12-23 Amsterdam Molecular Therapeutics B.V. Parvoviral capsid with incorporated Gly-Ala repeat region
US8945885B2 (en) 2008-07-03 2015-02-03 The Board Of Trustees Of The Leland Stanford Junior University Minicircle DNA vector preparations and methods of making and using the same
AU2009297243B2 (en) 2008-09-29 2015-10-29 Proyecto De Biomedicina Cima S.L. Porphobilinogen deaminase gene therapy
WO2010051367A1 (en) 2008-10-31 2010-05-06 The Trustees Of The University Of Pennsylvania Simian adenoviruses sadv-43, -45,-48,-49, and -50 and uses thereof
JP2012508591A (ja) 2008-11-14 2012-04-12 ライフ テクノロジーズ コーポレーション 細胞を操作するための組成物および方法
US9415121B2 (en) 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
WO2010093784A2 (en) 2009-02-11 2010-08-19 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
ES2742180T3 (es) 2009-03-04 2020-02-13 Deutsches Krebsforsch Proteína activadora del ensamblaje (AAP) y su uso para la fabricación de partículas de parvovirus que consisten esencialmente en VP3
US20120093775A1 (en) 2009-03-27 2012-04-19 Proyecto De Biomedicina Cima, S.L. Methods and compositions for the treatment of cirrhosis and liver fibrosis
EP2425000B1 (en) 2009-04-30 2019-02-20 The Trustees Of The University Of Pennsylvania Compositions for targeting conducting airway cells comprising adeno-associated virus constructs
EP2424980A1 (en) 2009-05-01 2012-03-07 Redbiotec AG Recombinant virus-like particles encoded by multi-gene vector
HRP20250897T1 (hr) 2009-05-02 2025-09-26 Genzyme Corporation Genska terapija za neurodegenerativne poremećaje
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
EP2435575A2 (en) 2009-05-28 2012-04-04 Deutsches Krebsforschungszentrum Modified aav capsid polypeptides
CA2762203A1 (en) 2009-05-29 2010-12-02 Soumitra Roy Simian adenovirus 41 and uses thereof
KR20180000341A (ko) 2009-06-16 2018-01-02 젠자임 코포레이션 재조합 ａａｖ 벡터에 대한 개선된 정제 방법
JP2014501097A (ja) 2009-07-06 2014-01-20 アルナイラム ファーマシューティカルズ， インコーポレイテッド 生物由来物質の産生を高めるための組成物及び方法
EP2292781A1 (en) 2009-08-17 2011-03-09 Genethon Baculovirus-based production of biopharmaceuticals free of contaminating baculoviral virions
WO2011038187A1 (en) 2009-09-25 2011-03-31 The Trustees Of The University Of Pennsylvania Controlled adeno-associated virus (aav) diversification and libraries prepared therefrom
KR20120089743A (ko) 2009-11-09 2012-08-13 제네포드 테라퓨틱스 에이비 생체 내에서 최적화된 뉴런 특이적 연속 도파 합성을 위한 신규한 바이러스 벡터 구성물
WO2011069529A1 (en) 2009-12-09 2011-06-16 Curevac Gmbh Mannose-containing solution for lyophilization, transfection and/or injection of nucleic acids
EP2524037B1 (en) 2010-01-12 2018-05-16 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
EP2528941A4 (en) 2010-01-25 2013-05-29 Univ New York FOR TRANSPORT STUDIES AND NEURONAL ADMINISTRATION, MANIPULATED RECOMBINANT DERIVATIVES FROM BOTULINUM NEUROTOXINES
US9169492B2 (en) 2010-02-05 2015-10-27 The University Of North Carolina At Chapel Hill Compositions and methods for enhanced parvovirus transduction
EP2545165B1 (en) 2010-03-11 2020-07-29 uniQure IP B.V. Mutated rep encoding sequences for use in aav production
US9546112B2 (en) 2010-03-22 2017-01-17 Association Institut De Myologie Methods of increasing efficiency of vector penetration of target tissue
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
WO2011126808A2 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
US9546369B2 (en) 2010-04-23 2017-01-17 University Of Massachusetts Multicistronic expression constructs
EP2561073B1 (en) 2010-04-23 2016-08-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
EP3444346B1 (en) 2010-04-23 2022-07-27 University of Massachusetts Aav-based treatment of cholesterol-related disorders
US9839696B2 (en) 2010-04-30 2017-12-12 City Of Hope Recombinant adeno-associated vectors for targeted treatment
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
EP2605798A1 (en) 2010-07-12 2013-06-26 Universidad Autònoma de Barcelona Gene therapy composition for use in diabetes treatment
US8808684B2 (en) 2010-09-10 2014-08-19 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Epidermal growth factor receptor (EGFR) and methods of use in adenoviral-associated virus type 6 (AAV6) transduction
CN103124787B (zh) 2010-10-05 2015-04-29 宝生物工程株式会社 制造病毒载体的方法
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
US9175280B2 (en) 2010-10-12 2015-11-03 Sangamo Biosciences, Inc. Methods and compositions for treating hemophilia B
WO2012057363A1 (ja) * 2010-10-27 2012-05-03 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
WO2012061786A1 (en) 2010-11-05 2012-05-10 Brandeis University Ice cleaved alpha-synuclein a biomarker
WO2012064920A1 (en) 2010-11-11 2012-05-18 University Of Miami Methods, compositions, cells, and kits for treating ischemic injury
AU2011332025B2 (en) 2010-11-23 2015-06-25 The Trustees Of The University Of Pennsylvania Subfamily E simian adenoviruses A1321, A1325, A1295, A1309 and A1322 and uses thereof
WO2012109570A1 (en) 2011-02-10 2012-08-16 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
TR201908020T4 (tr) 2011-02-17 2019-06-21 Univ Pennsylvania Doku özgüllüğünü değiştirmek ve aav9-aracılı gen transferini geliştirmek için bileşimler ve yöntemler.
GB201103062D0 (en) 2011-02-22 2011-04-06 Isis Innovation Method
US8865881B2 (en) 2011-02-22 2014-10-21 California Institute Of Technology Delivery of proteins using adeno-associated virus (AAV) vectors
US20120220648A1 (en) * 2011-02-24 2012-08-30 National Taiwan University Hospital Method of Treating Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency Using Adeno-Associated Virus (AAV)-AADC Vector
EP2500434A1 (en) 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
CN103476399A (zh) 2011-04-18 2013-12-25 独立行政法人国立精神·神经医疗研究中心 药剂递送粒子及其制造方法
WO2012145572A1 (en) 2011-04-20 2012-10-26 The Trustees Of The University Of Pennsylvania Regimens and compositions for aav-mediated passive immunization of airborne pathogens
EP3318635A1 (en) 2011-04-21 2018-05-09 University of Massachusetts Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
CN105755044A (zh) 2011-04-22 2016-07-13 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
US9249425B2 (en) 2011-05-16 2016-02-02 The Trustees Of The University Of Pennslyvania Proviral plasmids and production of recombinant adeno-associated virus
WO2012159006A2 (en) 2011-05-18 2012-11-22 University Of Florida Research Foundation, Inc. Polypeptides and vectors for targeting her2/neu expressing cells and uses thereof
US20120295960A1 (en) 2011-05-20 2012-11-22 Oxford Biomedica (Uk) Ltd. Treatment regimen for parkinson's disease
KR20140023326A (ko) 2011-06-06 2014-02-26 비오까르띠 에스아 이온성 계면활성제에 의한 세포의 선택적 용해
JP6122430B2 (ja) 2011-07-27 2017-04-26 ジェネトン 改善されたバキュロウイルス発現系
JP6198278B2 (ja) 2011-09-08 2017-09-20 ユニキュアー アイピー ビー．ブイ． Ａａｖ調製物からの混入ウイルスの除去
WO2013036889A1 (en) 2011-09-09 2013-03-14 University Of Washington Retrograde transport peptide and use of same for delivery to central nervous system
GB201118636D0 (en) 2011-10-28 2011-12-07 Oxford Biomedica Ltd Nucleotide sequence
CA2853482C (en) 2011-10-28 2020-05-19 Joshua Grieger Cell line for production of adeno-associated virus
HK1202434A1 (en) 2011-11-22 2015-10-02 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
WO2013078199A2 (en) 2011-11-23 2013-05-30 Children's Medical Center Corporation Methods for enhanced in vivo delivery of synthetic, modified rnas
WO2013103896A1 (en) 2012-01-06 2013-07-11 Mayo Foundation For Medical Education And Research Treating cardiovascular or renal diseases
CA2864100A1 (en) 2012-02-14 2013-08-22 The Regents Of The University Of California Systemic delivery and regulated expression of paracrine genes for cardiovascular diseases and other conditions
MX367100B (es) 2012-02-17 2019-08-05 The Children´S Hospital Of Philadelphia Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos.
NZ701693A (en) 2012-04-18 2017-02-24 The Children’S Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using aav capsid variants
EP2660325A3 (en) 2012-05-02 2014-02-12 Christian Medical College AAV vectors and corresponding nucleotide sequences and methods
US9163259B2 (en) 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
KR102108586B1 (ko) 2012-05-08 2020-05-07 머크 샤프 앤드 돔 코포레이션 투과성 글리코시다제 억제제 및 그의 용도
WO2013170078A1 (en) 2012-05-09 2013-11-14 Oregon Health & Science University Adeno associated virus plasmids and vectors
TWI698240B (zh) 2012-05-15 2020-07-11 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
SG11201407343XA (en) 2012-05-18 2014-12-30 Univ Pennsylvania Subfamily e simian adenoviruses a1302, a1320, a1331 and a1337 and uses thereof
RU2679843C2 (ru) 2012-07-06 2019-02-13 Юниверсити Оф Айова Рисерч Фаундейшн Векторные композиции с модифицированным аденоассоциированным вирусом
KR101738438B1 (ko) 2012-07-06 2017-05-22 다카라 바이오 가부시키가이샤 아데노-관련 바이러스 벡터의 생산세포
AU2013296425B2 (en) 2012-08-01 2018-06-07 Nationwide Children's Hospital Intrathecal delivery of recombinant adeno-associated virus 9
WO2014036468A2 (en) 2012-08-31 2014-03-06 The United States Of America, As Represented By The Secretary, Department Of Health & Human Services Aav mediated aquaporin gene transer to treat sjogren's syndrome
EP2900686B1 (en) 2012-09-28 2020-06-10 The University of North Carolina At Chapel Hill Aav vectors targeted to oligodendrocytes
WO2014071042A1 (en) 2012-10-31 2014-05-08 The Trustees Of Columbia University In The City Of New York Methods for identifying candidates for the treatment of neurodegenerative diseases
EP3031921B1 (en) 2012-12-12 2025-03-12 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
US9938541B2 (en) 2012-12-25 2018-04-10 Takara Bio Inc. AAV variant
EP3521420A1 (en) 2013-01-08 2019-08-07 Genzyme Corporation Use of inos inhibitors to increase viral yield in culture
US9066966B2 (en) 2013-02-01 2015-06-30 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia
US9567376B2 (en) 2013-02-08 2017-02-14 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
CA2905952A1 (en) 2013-03-13 2014-10-02 The Children's Hospital Of Philadelphia Adeno-associated virus vectors and methods of use thereof
WO2014143932A1 (en) 2013-03-15 2014-09-18 The University Of North Carolina At Chapel Hill Synthetic adeno-associated virus inverted terminal repeats
HK1220488A1 (zh) 2013-03-15 2017-05-05 The Children's Hospital Of Philadelphia 含有填充者/填充物多核苷酸序列的载体及其使用方法
WO2014168953A1 (en) 2013-04-08 2014-10-16 University Of Iowa Research Foundation Chimeric adeno-associated virus/ bocavirus parvovirus vector
EP2792742A1 (en) 2013-04-17 2014-10-22 Universitätsklinikum Hamburg-Eppendorf (UKE) Gene-therapy vectors for treating cardiomyopathy
AU2014255665B2 (en) 2013-04-18 2018-08-02 Fondazione Telethon Effective delivery of large genes by dual AAV vectors
MX388221B (es) 2013-04-20 2025-03-19 Res Institute At Nationwide Children´S Hospital ADMINISTRACIÓN DE VIRUS ADENO-ASOCIADO RECOMBINANTE DE CONSTRUCCIONES DE POLINUCLÉOTIDOS U7snRNA DIRIGIDA AL EXÓN 2.
WO2015012924A2 (en) 2013-04-29 2015-01-29 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof
CN115120746A (zh) 2013-05-15 2022-09-30 明尼苏达大学董事会 腺相关病毒介导的基因向中枢神经系统转移
WO2014186746A1 (en) 2013-05-16 2014-11-20 University Of Florida Research Foundation, Inc. HAIRPIN mRNA ELEMENTS AND METHODS FOR THE REGULATION OF PROTEIN TRANSLATION
EP3003391B1 (en) 2013-05-31 2021-09-22 The Regents of The University of California Adeno-associated virus variants and methods of use thereof
WO2014201308A1 (en) 2013-06-12 2014-12-18 Washington University Endothelial-targeted adenoviral vectors, methods and uses therefor
US20160122727A1 (en) 2013-06-13 2016-05-05 Shire Human Genetic Therapies, Inc. Messenger rna based viral production
IL297919A (en) 2013-07-22 2023-01-01 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
EP3024497B1 (en) 2013-07-26 2021-01-13 University of Iowa Research Foundation Methods and compositions for treating brain diseases
ITTO20130669A1 (it) 2013-08-05 2015-02-06 Consiglio Nazionale Ricerche Vettore adeno-associato ricombinante muscolo-specifico e suo impiego nel trattamento di patologie muscolari
WO2015031686A1 (en) 2013-08-30 2015-03-05 Amgen Inc. High titer recombinant aav vector production in adherent and suspension cells
EP3044231B1 (en) 2013-09-12 2020-08-05 BioMarin Pharmaceutical Inc. Aav vectors comprising a gene encoding factor viii
ES2739288T3 (es) 2013-09-13 2020-01-30 California Inst Of Techn Recuperación selectiva
WO2015044292A1 (en) 2013-09-26 2015-04-02 Universitat Autonoma De Barcelona Gene therapy compositions for use in the prevention and/or treatment of non-alcoholic fatty liver disease
WO2015048534A1 (en) 2013-09-26 2015-04-02 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
CN106232618A (zh) 2013-10-11 2016-12-14 马萨诸塞眼科耳科诊所 预测祖先病毒序列的方法及其用途
CN105745326A (zh) 2013-10-24 2016-07-06 优尼科Ip有限公司 用于基因治疗神经疾病的aav-5假型载体
US20160272687A1 (en) 2013-11-08 2016-09-22 The Board Of Trustees Of The University Of Arkansas Adeno-associated virus "x" oncogene
AU2014354839B2 (en) 2013-11-26 2020-06-04 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Adeno-associated virus vectors for treatment of glycogen storage disease
CN105765066B (zh) 2013-11-29 2018-12-14 宝生物工程株式会社 用于定量腺伴随病毒的方法
JP6707028B2 (ja) 2013-12-09 2020-06-10 ベイラー カレッジ オブ メディスンＢａｙｌｏｒ Ｃｏｌｌｅｇｅ Ｏｆ Ｍｅｄｉｃｉｎｅ 心筋細胞新生におけるｈｉｐｐｏ及びジストロフィン複合体シグナル伝達
JP6712948B2 (ja) 2013-12-12 2020-06-24 ザ・ブロード・インスティテュート・インコーポレイテッド 組成物、及びヌクレオチドリピート障害におけるｃｒｉｓｐｒ−ｃａｓ系の使用方法
GB201322798D0 (en) 2013-12-20 2014-02-05 Oxford Biomedica Ltd Production system
EP3092493B1 (en) 2014-01-09 2019-04-17 Genethon Methods for diagnosing or monitoring muscular dystrophies
WO2015106273A2 (en) 2014-01-13 2015-07-16 Trustees Of Boston University Methods and assays relating to huntingtons disease and parkinson's disease
GB201401707D0 (en) 2014-01-31 2014-03-19 Sec Dep For Health The Adeno-associated viral vectors
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
WO2015124546A1 (en) 2014-02-19 2015-08-27 Fundación Centro Nacional De Investigaciones Cardiovasculares Carlos Iii- Cnic Aav vectors for the treatment of ischemic and non-ischemic heart disease
SG11201606824VA (en) 2014-02-19 2016-09-29 Hoffmann La Roche Blood brain barrier shuttle
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
US20170007720A1 (en) 2014-02-21 2017-01-12 University Of Florida Research Foundation, Inc. Methods and compositions for gene delivery to on bipolar cells
US10272117B2 (en) 2014-02-24 2019-04-30 Celgene Corporation Methods of using an activator of cereblon for neural cell expansion and the treatment of central nervous system disorders
WO2015131076A1 (en) 2014-02-27 2015-09-03 Yale University Nlrp6 inflammasome intestinal epithelium mucus secretion
EP3113787B1 (en) 2014-03-04 2019-12-04 University of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
MX2016011517A (es) 2014-03-07 2017-04-13 Univ Arizona Peptidos de crmp2 no narcoticos que seleccionan como diana canales de sodio para el dolor cronico.
SMT202000531T1 (it) 2014-03-09 2020-11-10 Univ Pennsylvania Composizioni utili in trattamento di deficit di ornitina transcarbamilasi(otc)
CA2942289C (en) 2014-03-10 2024-05-21 Uniqure Ip B.V. Further improved aav vectors produced in insect cells
EP3126506A4 (en) 2014-04-03 2017-11-22 Braingene AB Gene expression system and regulation thereof
WO2015157070A2 (en) 2014-04-09 2015-10-15 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating cystic fibrosis
EP2933335A1 (en) 2014-04-18 2015-10-21 Genethon A method of treating peripheral neuropathies and motor neuron diseases
WO2015164786A1 (en) 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
EP3134431B1 (en) 2014-04-25 2021-04-07 The Trustees Of The University Of Pennsylvania Ldlr variants and their use in compositions for reducing cholesterol levels
EP3142706A1 (en) 2014-05-16 2017-03-22 Vrije Universiteit Brussel Genetic correction of myotonic dystrophy type 1
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
US11207424B2 (en) 2014-06-13 2021-12-28 Mayo Foundation For Medical Education And Research Methods and materials for increasing viral vector infectivity
US10781459B2 (en) 2014-06-20 2020-09-22 University Of Florida Research Foundation, Incorporated Methods of packaging multiple adeno-associated virus vectors
EP2960336A1 (en) 2014-06-27 2015-12-30 Genethon Efficient systemic treatment of dystrophic muscle pathologies
WO2016004319A1 (en) 2014-07-02 2016-01-07 University Of Florida Research Foundation, Inc. Compositions and methods for purifying recombinant adeno-associated virus
US10023846B2 (en) 2014-07-10 2018-07-17 Takara Bio Inc. Production method for non-enveloped virus particles
WO2016019144A2 (en) 2014-07-30 2016-02-04 Sangamo Biosciences, Inc. Gene correction of scid-related genes in hematopoietic stem and progenitor cells
US10392622B2 (en) 2014-08-01 2019-08-27 The Trustees Of The University Of Pennsylvania Compositions and methods for self-regulated inducible gene expression
EP3200830B1 (en) 2014-10-03 2020-09-09 University of Massachusetts High efficiency library-identified aav vectors
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
US10842886B2 (en) 2014-10-10 2020-11-24 Research Institute At Nationwide Children's Hospital Guided injections for AAV gene transfer to muscle
CN107073051B (zh) 2014-10-21 2021-08-24 马萨诸塞大学 重组aav变体及其用途
US10907130B2 (en) 2014-11-05 2021-02-02 Research Institute At Nationwide Children's Hospital Methods and materials for producing recombinant viruses in eukaryotic microalgae
CN107428820B (zh) 2014-11-19 2022-03-22 阿克松神经系统科学公司 在阿尔茨海默氏病中的人源化tau抗体
US10900053B2 (en) 2014-11-21 2021-01-26 University Of Florida Research Foundation, Incorporated Genome-modified recombinant adeno-associated virus vectors
EP4344741A3 (en) 2014-11-21 2024-08-28 The University of North Carolina at Chapel Hill Aav vectors targeted to the central nervous system
HUE046643T2 (hu) 2014-11-28 2021-11-29 Uniqure Ip Bv DNS szennyezõdések parvovirális virionokat tartalmazó készítményben
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
US10907176B2 (en) 2015-01-14 2021-02-02 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
US20180008727A1 (en) 2015-01-30 2018-01-11 The Regents Of The University Of California Spinal subpial gene delivery system
US20180030096A1 (en) 2015-02-03 2018-02-01 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
CN107530447A (zh) 2015-02-10 2018-01-02 建新公司 病毒颗粒至纹状体和皮质的增强递送
WO2016137949A1 (en) 2015-02-23 2016-09-01 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
KR20240119152A (ko) 2015-03-10 2024-08-06 더 트러스티스 오브 컬럼비아 유니버시티 인 더 시티 오브 뉴욕 재조합 glut1 아데노-관련 바이러스 벡터 작제물 및 glut1 발현을 회복하기 위한 관련된 방법
WO2016154055A1 (en) 2015-03-20 2016-09-29 Bluebird Bio, Inc. Vector formulations
JP6836999B2 (ja) 2015-03-24 2021-03-03 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアＴｈｅ Ｒｅｇｅｎｔｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｃａｌｉｆｏｒｎｉａ アデノ随伴ウイルス変異体及びその使用方法
US20160319278A1 (en) 2015-04-03 2016-11-03 University Of Massachusetts Fully stabilized asymmetric sirna
US10081659B2 (en) 2015-04-06 2018-09-25 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Adeno-associated vectors for enhanced transduction and reduced immunogenicity
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
EP3283126B1 (en) 2015-04-16 2019-11-06 Emory University Recombinant promoters and vectors for protein expression in liver and use thereof
EP3285788B1 (en) 2015-04-23 2024-12-18 University of Massachusetts Modulation of aav vector transgene expression
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
WO2016179038A1 (en) 2015-05-01 2016-11-10 Spark Therapeutics, Inc. ADENO-ASSOCIATED VIRUS-MEDIATED CRISPR-Cas9 TREATMENT OF OCULAR DISEASE
EP3291765A4 (en) 2015-05-07 2019-01-23 Massachusetts Eye & Ear Infirmary METHOD FOR THE DELIVERY OF AN ACTIVE SUBSTANCE TO THE EYE
US9682193B2 (en) 2015-05-11 2017-06-20 Alcyone Lifesciences, Inc. Drug delivery systems and methods
JP6805174B2 (ja) 2015-05-12 2020-12-23 アメリカ合衆国 神経成長因子シグナルペプチド及び副甲状腺ホルモンを含むａａｖ分離株及び融合タンパク質
US20170067028A1 (en) 2015-05-15 2017-03-09 Douglas J. Ballon Radiolabeling of adeno associated virus
WO2016191418A1 (en) 2015-05-26 2016-12-01 Salk Institute For Biological Studies Motor neuron-specific expression vectors
US10214731B2 (en) 2015-05-28 2019-02-26 Cornell University Adeno-associated virus mediated delivery of C1E1 as a therapy for angioedema
WO2016196507A1 (en) 2015-05-29 2016-12-08 University Of Iowa Research Foundation Methods of delivery of transgenes for treating brain diseases
MX2017015373A (es) 2015-05-29 2018-07-06 Univ Pennsylvania COMPOSICIONES Y Mí‰TODOS PARA LA DEGRADACIí“N DE PROTEíNAS MAL PLEGADAS.
MX390848B (es) 2015-06-23 2025-03-21 Childrens Hospital Philadelphia Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos.
WO2017004514A1 (en) 2015-07-02 2017-01-05 University Of Florida Research Foundation, Inc. Recombinant adeno-associated virus vectors to target medullary thyroid carcinoma
ES2895652T3 (es) 2015-07-07 2022-02-22 Inst Nat Sante Rech Med Métodos y composiciones farmacéuticas para expresar un polinucleótido de interés en el sistema nervioso periférico de un sujeto
US20170007669A1 (en) 2015-07-07 2017-01-12 Mayo Foundation For Medical Education And Research Peptide-mediated delivery of active agents across the blood-brain barrier
WO2017015102A1 (en) 2015-07-17 2017-01-26 The Trustees Of The University Of Pennsylvania Compositions and methods for achieving high levels of transduction in human liver cells
WO2017019876A1 (en) 2015-07-28 2017-02-02 University Of Massachusetts Transgenic expression of dnase i in vivo delivered by an adeno-associated virus vector
CN108603235A (zh) 2015-07-30 2018-09-28 马萨诸塞眼科耳科诊所 祖先病毒序列及其用途
US20190000940A1 (en) 2015-07-31 2019-01-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of aadc deficiency
WO2017024198A1 (en) 2015-08-06 2017-02-09 The Trustees Of The University Of Pennsylvania Glp-1 and use thereof in compositions for treating metabolic diseases
TW202446416A (zh) 2015-09-24 2024-12-01 美商拜奧馬林製藥公司 腺相關病毒因子viii載體、相關病毒粒子及包含其之治療調配物
ES2865487T3 (es) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
EP4530350A3 (en) 2015-10-09 2025-06-25 Genzyme Corporation Improved flare (flow cytometry attenuated reporter expression) technology for rapid bulk sorting
US10123969B2 (en) 2015-10-15 2018-11-13 Wisconsin Alumni Research Foundation Osmotic enhancement of drug/therapeutic delivery to the brain following infusion or injection into the cerebrospinal fluid
WO2017070516A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
US12188037B2 (en) 2015-10-22 2025-01-07 University Of Florida Research Foundation, Incorporated Synthetic combinatorial AAV3 capsid library
EP3364997B1 (en) 2015-10-22 2024-01-17 University of Massachusetts Aspartoacylase gene therapy in the treatment of canavan disease
KR102738860B1 (ko) 2015-10-23 2024-12-04 유니버시티 오브 아이오와 리써치 파운데이션 리소좀 축적 질환 (lsd)을 갖는 포유동물의 치료 방법에 사용하기 위한 aav 입자를 포함하는 약제학적 조성물
WO2017075335A1 (en) 2015-10-28 2017-05-04 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
CN108779167A (zh) 2015-10-28 2018-11-09 宾夕法尼亚州大学信托人 鞘内施用腺伴随病毒载体用于基因治疗
CN118725134A (zh) 2015-11-08 2024-10-01 豪夫迈·罗氏有限公司 筛选多特异性抗体的方法
US10633662B2 (en) 2015-11-10 2020-04-28 The Board Of Trustees Of The Leland Stanford Junior University Methods and compositions for modulating AAV infection
EP3384015A4 (en) 2015-12-01 2019-05-29 Spark Therapeutics, Inc. SCALABLE PROCESS FOR THE PREPARATION OF A RECOMBINANT ADENO ASSOCIATED VIRAL VECTOR IN A SERUM-FREE SUSPENSION CELL CULTURE SYSTEM SUITABLE FOR THE CLINICAL APPLICATION
US20170151416A1 (en) 2015-12-01 2017-06-01 Invivo Therapeutics Corporation Methods and Systems for Delivery of a Trail of a Therapeutic Substance into an Anatomical Space
EP3384034B1 (en) 2015-12-02 2020-07-08 The Board of Trustees of the Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
US10406244B2 (en) 2015-12-02 2019-09-10 The Board Of Trustees Of The Leland Stanford Junior University AAV vectors with expanded packaging capacity
EP3383896A1 (en) 2015-12-03 2018-10-10 Genethon Compositions and methods for improving viral vector efficiency
US11028372B2 (en) 2015-12-11 2021-06-08 The Trustees Of The University Of Pennsylvania Scalable purification method for AAVRH10
EP3387138B1 (en) 2015-12-11 2022-01-26 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
ES2934848T3 (es) 2015-12-11 2023-02-27 Univ Pennsylvania Método de purificación escalable para AAV8
KR102423442B1 (ko) 2015-12-11 2022-07-20 캘리포니아 인스티튜트 오브 테크놀로지 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드
JP7406783B2 (ja) 2015-12-14 2023-12-28 ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質
WO2017112948A1 (en) 2015-12-24 2017-06-29 University Of Florida Research Foundation, Inc. Improved aav production using suspension adapted cells
RU2771490C2 (ru) 2016-01-13 2022-05-05 Мериал, Инк. Рекомбинантные aav векторы, экспрессирующие остеопротективные гены, включая has2 и лубрицин, пригодные при лечении остеоартрита и сходных заболеваний суставов у млекопитающих
JP6924441B2 (ja) 2016-01-15 2021-08-25 学校法人自治医科大学 てんかん治療のためのアデノ随伴ウイルスビリオン
CA3011939A1 (en) 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
EP3411484B1 (en) 2016-02-05 2023-10-04 Emory University Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid
WO2017139381A1 (en) 2016-02-08 2017-08-17 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
JP6947739B2 (ja) 2016-02-16 2021-10-13 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 既存のヒト中和抗体に対して耐性である新規組換えアデノ随伴ウイルスカプシド
WO2017147477A1 (en) 2016-02-26 2017-08-31 University Of Florida Research Foundation, Inc. Aav heparin mutants that display significantly improved eye and brain transduction
US20190328906A1 (en) 2016-03-02 2019-10-31 The Children's Hospital Of Philadelphia Therapy for frontotemporal dementia
MX2018010633A (es) 2016-03-03 2019-06-13 Univ Massachusetts Acido desoxirribonucleico (adn) lineal duplex de extremos cerrados para transferencia genica no viral.
CA3016985C (en) 2016-03-07 2023-07-04 University Of Iowa Research Foundation Aav-mediated expression using a synthetic promoter and enhancer
CN109476716A (zh) 2016-03-23 2019-03-15 加利福尼亚大学董事会 治疗线粒体障碍的方法
WO2017165859A1 (en) 2016-03-24 2017-09-28 Research Institute At Nationwide Children's Hospital Modified viral capsid proteins
WO2017172733A1 (en) 2016-03-28 2017-10-05 The Regents Of The University Of California Anti-ryk antibodies and methods of using the same
FI3436593T3 (fi) 2016-03-28 2023-03-16 Ultragenyx Pharmaceutical Inc Adenoviruksen lämpöinaktivointimenetelmiä
WO2017173043A1 (en) 2016-03-30 2017-10-05 Spark Therapeutics, Inc. Cell line for recombinant protein and/or viral vector production
LT3436051T (lt) 2016-03-31 2021-11-25 Spark Therapeutics, Inc. Kolonėlėmis pagrįstas visiškai keičiamo masto raav gavimo būdas
SG11201808812RA (en) 2016-04-15 2018-11-29 Univ Pennsylvania Novel aav8 mutant capsids and compositions containing same
KR102598289B1 (ko) 2016-04-16 2023-11-06 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 바큘로바이러스 시스템-생산된 재조합 아데노-연관 바이러스의 생물학적 효력을 증진시키는 방법
JP7496667B2 (ja) 2016-04-21 2024-06-07 ビロベク，インコーポレイテッド 昆虫細胞中でのａａｖ生成、方法およびその組成物
EP3235516B1 (en) 2016-04-22 2019-06-26 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts Universitätsmedizin Regulatable adeno-associated virus (aav) vector
EP3449250B1 (en) 2016-04-28 2020-11-04 Indiana University Research & Technology Corporation Methods and compositions for resolving components of a virus preparation
AU2017261249B2 (en) 2016-05-03 2021-05-06 Children's Medical Research Institute Adeno-associated virus polynucleotides, polypeptides and virions
US11866462B2 (en) 2016-05-04 2024-01-09 Oregon Health & Science University Recombinant adeno-associated viral vectors
US10898585B2 (en) 2017-04-14 2021-01-26 Ptc Therapeutics .Inc. Gene therapy for AADC deficiency
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
KR20210019996A (ko) 2018-05-15 2021-02-23 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 조성물 및 방법

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016073693A2 (en) * 2014-11-05 2016-05-12 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
DATABASE Genbank [o] 25 August 2016 (2016-08-25), "Adeno-associated virus - 2 Homo sapiens DNA fragment containing the 5' cellular/adeno-associated viral junction", Database accession no. AH002785.2 *
DATABASE Genbank [o] 30 September 2012 (2012-09-30), "Insertion vector scAAV-CMV-GFP, complete sequence", Database accession no. JX445134.1 *

Cited By (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11975056B2 (en) 2014-11-05 2024-05-07 Voyager Therapeutics, Inc. AADC polynucleotides for the treatment of Parkinson's disease
US12138318B2 (en) 2015-05-11 2024-11-12 Ucl Business Ltd Capsid
US10414803B2 (en) 2015-05-11 2019-09-17 Ucl Business Plc Capsid
US11525139B2 (en) 2016-08-23 2022-12-13 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
US12264317B2 (en) 2016-08-23 2025-04-01 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
US12071627B2 (en) 2016-08-23 2024-08-27 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
US11993777B2 (en) 2016-08-23 2024-05-28 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
US11781145B2 (en) 2016-08-23 2023-10-10 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
US11759506B2 (en) 2017-06-15 2023-09-19 Voyager Therapeutics, Inc. AADC polynucleotides for the treatment of Parkinson's disease
CN112424359A (zh) * 2018-05-15 2021-02-26 沃雅戈治疗公司 用于治疗帕金森氏病的组合物和方法
US12319929B2 (en) 2018-05-15 2025-06-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of Parkinson's disease
WO2019222328A1 (en) * 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
WO2019241486A1 (en) 2018-06-13 2019-12-19 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
WO2020023612A1 (en) 2018-07-24 2020-01-30 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
WO2020072849A1 (en) 2018-10-04 2020-04-09 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
WO2020072844A1 (en) 2018-10-05 2020-04-09 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding aav production proteins
WO2020081490A1 (en) 2018-10-15 2020-04-23 Voyager Therapeutics, Inc. EXPRESSION VECTORS FOR LARGE-SCALE PRODUCTION OF rAAV IN THE BACULOVIRUS/Sf9 SYSTEM
EP3943503A4 (en) * 2019-01-30 2023-10-18 Shanghaitech University Adeno-associated virus having variant capsid protein and use thereof
WO2020223274A1 (en) 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS
WO2021007382A1 (en) * 2019-07-09 2021-01-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
WO2021030125A1 (en) 2019-08-09 2021-02-18 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
WO2021041485A1 (en) 2019-08-26 2021-03-04 Voyager Therapeutics, Inc. Controlled expression of viral proteins
JP2023506007A (ja) * 2019-12-12 2023-02-14 メイラグティーエックス ユーケー アイアイ リミティド パーキンソン病を治療するための方法
JP7758671B2 (ja) 2019-12-12 2025-10-22 メイラグティーエックス ユーケー アイアイ リミティド パーキンソン病を治療するための方法
WO2021119615A1 (en) * 2019-12-12 2021-06-17 Meiragtx Uk Ii Limited Methods for treating parkinson's disease
US11807867B2 (en) 2020-02-21 2023-11-07 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
US12305191B2 (en) 2020-02-21 2025-05-20 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
US12077773B2 (en) 2020-02-21 2024-09-03 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
WO2022032153A1 (en) 2020-08-06 2022-02-10 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
WO2022187548A1 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022187473A2 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
US12116385B2 (en) 2021-10-08 2024-10-15 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
US12331081B2 (en) 2021-10-08 2025-06-17 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
US12331082B2 (en) 2021-10-08 2025-06-17 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
US12528841B2 (en) 2021-10-08 2026-01-20 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
WO2023093905A1 (zh) 2021-11-29 2023-06-01 上海瑞宏迪医药有限公司 Aadc、gdnf多核苷酸及其用于治疗帕金森病
WO2023155918A1 (zh) 2022-02-21 2023-08-24 上海瑞宏迪医药有限公司 Vegf结合分子及其医药用途
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2025040166A1 (zh) 2023-08-23 2025-02-27 上海瑞宏迪医药有限公司 药物组合物及其用途
WO2025051805A1 (en) * 2023-09-04 2025-03-13 Uniqure Biopharma B.V. Novel neurotropic recombinant adeno-associated virus particles

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