BR112022016936A2 - Terapia gênica - Google Patents

Terapia gênica

Info

Publication number
BR112022016936A2
BR112022016936A2 BR112022016936A BR112022016936A BR112022016936A2 BR 112022016936 A2 BR112022016936 A2 BR 112022016936A2 BR 112022016936 A BR112022016936 A BR 112022016936A BR 112022016936 A BR112022016936 A BR 112022016936A BR 112022016936 A2 BR112022016936 A2 BR 112022016936A2
Authority
BR
Brazil
Prior art keywords
gene therapy
vector
kidney
promoter
nphs2
Prior art date
Application number
BR112022016936A
Other languages
English (en)
Inventor
R Foster Rebecca
Ahson Saleem-Uddin Moin
Iain Welsh Gavin
Original Assignee
Univ Bristol
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Univ Bristol filed Critical Univ Bristol
Publication of BR112022016936A2 publication Critical patent/BR112022016936A2/pt

Links

Classifications

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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

TERAPIA GÊNICA. A presente invenção fornece uma terapia gênica de vetor de vírus adeno-associado (AAV) compreendendo um transgene de fator de crescimento endotelial vascular (VEGF)C; e promotor mínimo de nefrina NPHS1 ou promotor de podocina NPHS2. O vetor da terapia gênica pode ser usado para direcionar podócitos dentro do glomérulo do rim a fim de tratar ou prevenir doenças renais, tal como a doença renal diabética.
BR112022016936A 2020-03-04 2021-03-04 Terapia gênica BR112022016936A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GBGB2003109.2A GB202003109D0 (en) 2020-03-04 2020-03-04 Gene therapy
PCT/GB2021/050537 WO2021176220A1 (en) 2020-03-04 2021-03-04 Gene therapy

Publications (1)

Publication Number Publication Date
BR112022016936A2 true BR112022016936A2 (pt) 2022-11-22

Family

ID=70278767

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022016936A BR112022016936A2 (pt) 2020-03-04 2021-03-04 Terapia gênica

Country Status (11)

Country Link
US (1) US20230101788A1 (pt)
EP (1) EP4114957A1 (pt)
JP (1) JP2023515792A (pt)
CN (1) CN115315517A (pt)
AU (1) AU2021231959A1 (pt)
BR (1) BR112022016936A2 (pt)
CA (1) CA3170657A1 (pt)
GB (1) GB202003109D0 (pt)
IL (1) IL296016A (pt)
MX (1) MX2022010934A (pt)
WO (1) WO2021176220A1 (pt)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB202217332D0 (en) * 2022-11-18 2023-01-04 Univ Bristol Methods

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR2804127B1 (fr) * 2000-01-20 2004-12-24 Inst Nat Sante Rech Med Gene srn1 implique dans le syndrome nephrotique cortico- resistant, proteine codee par ce gene et utilisations diagnostiques et therapeutiques
WO2002007757A2 (en) * 2000-07-21 2002-01-31 Lue Tom F Prevention and treatment of sexual arousal disorders
US20050043235A1 (en) * 2003-06-12 2005-02-24 Kari Alitalo Use of VEGF-C or VEGF-D in reconstructive surgery
US8852936B2 (en) 2012-09-21 2014-10-07 Laurantis Pharma Oy Autologous lymph node transfer in combination with VEGF-C or VEGF-D growth factor therapy to treat lymphedema and to improve reconstructive surgery
TW201506036A (zh) 2013-08-14 2015-02-16 Laurantis Pharma Oy Vegf-c及ccbe1之治療用途
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
WO2018204764A1 (en) * 2017-05-05 2018-11-08 Camp4 Therapeutics Corporation Identification and targeted modulation of gene signaling networks
JP7389980B2 (ja) * 2018-12-06 2023-12-01 国立大学法人 琉球大学 ヒト膵臓組織特異的幹／前駆細胞の人工作製方法
GB201900702D0 (en) 2019-01-18 2019-03-06 Univ Bristol Therapy
EP4126910A1 (en) * 2020-04-01 2023-02-08 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
GB202103470D0 (en) * 2021-03-12 2021-04-28 Univ Bristol Promoter

Also Published As

Publication number Publication date
WO2021176220A1 (en) 2021-09-10
AU2021231959A1 (en) 2022-09-15
IL296016A (en) 2022-10-01
GB202003109D0 (en) 2020-04-15
CN115315517A (zh) 2022-11-08
JP2023515792A (ja) 2023-04-14
CA3170657A1 (en) 2021-09-10
US20230101788A1 (en) 2023-03-30
EP4114957A1 (en) 2023-01-11
MX2022010934A (es) 2022-11-09

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