CN114126665B - 眼底黄色斑点症(abca4)的基因疗法 - Google Patents

眼底黄色斑点症(abca4)的基因疗法 Download PDF

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Publication number
CN114126665B
CN114126665B CN202080044221.2A CN202080044221A CN114126665B CN 114126665 B CN114126665 B CN 114126665B CN 202080044221 A CN202080044221 A CN 202080044221A CN 114126665 B CN114126665 B CN 114126665B
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CN114126665A (zh
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H·康纳
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University of Massachusetts Amherst
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University of Massachusetts Amherst
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    • 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
    • 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
    • A61K48/0075—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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
CN202080044221.2A 2019-04-19 2020-04-16 眼底黄色斑点症(abca4)的基因疗法 Active CN114126665B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962836493P 2019-04-19 2019-04-19
US62/836,493 2019-04-19
PCT/US2020/028504 WO2020214809A2 (en) 2019-04-19 2020-04-16 Gene therapies for stargardt disease (abca4)

Publications (2)

Publication Number Publication Date
CN114126665A CN114126665A (zh) 2022-03-01
CN114126665B true CN114126665B (zh) 2025-04-25

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CN202080044221.2A Active CN114126665B (zh) 2019-04-19 2020-04-16 眼底黄色斑点症(abca4)的基因疗法

Country Status (7)

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US (2) US12516097B2 (https=)
EP (1) EP3955973A4 (https=)
JP (1) JP2022529782A (https=)
CN (1) CN114126665B (https=)
AU (1) AU2020257392A1 (https=)
CA (1) CA3137135A1 (https=)
WO (1) WO2020214809A2 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3137135A1 (en) 2019-04-19 2020-10-22 University Of Massachusetts Gene therapies for stargardt disease (abca4)
GB2700048B (en) 2019-09-18 2026-03-04 Aldevron Llc synthetic dna vectors and method of use
CN116234917A (zh) 2020-07-27 2023-06-06 安杰瑞姆生物科学公司 Dna分子组合物及其制备方法和使用方法
KR20230161941A (ko) * 2021-01-22 2023-11-28 코넬 유니버시티 리포푸신 세포독성으로부터 보호하는 화합물의 식별을위한 조성물 및 방법
CA3211687A1 (en) * 2021-03-19 2022-09-22 Ozan ALKAN Non-viral dna vectors and uses thereof for expressing pfic therapeutics
EP4330409A4 (en) 2021-04-26 2025-04-09 University of Massachusetts GENE THERAPIES FOR STARGARDT'S DISEASE (ABCA4)
EP4334447A1 (en) * 2021-05-07 2024-03-13 UCL Business Ltd Abca4 genome editing
CN115074369B (zh) * 2022-06-24 2023-10-20 成都金唯科生物科技有限公司 表达全长abca4基因的腺相关病毒载体及应用

Family Cites Families (19)

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US5549910A (en) 1989-03-31 1996-08-27 The Regents Of The University Of California Preparation of liposome and lipid complex compositions
JP3218637B2 (ja) 1990-07-26 2001-10-15 大正製薬株式会社 安定なリポソーム水懸濁液
JP2958076B2 (ja) 1990-08-27 1999-10-06 株式会社ビタミン研究所 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法
US5399363A (en) 1991-01-25 1995-03-21 Eastman Kodak Company Surface modified anticancer nanoparticles
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
US5741516A (en) 1994-06-20 1998-04-21 Inex Pharmaceuticals Corporation Sphingosomes for enhanced drug delivery
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
US5738868A (en) 1995-07-18 1998-04-14 Lipogenics Ltd. Liposome compositions and kits therefor
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
US7943374B2 (en) * 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
US20090214478A1 (en) 2008-02-21 2009-08-27 Alberto Auricchio Method of treating ocular diseases by gene therapy
WO2009134418A2 (en) * 2008-04-30 2009-11-05 Fox Chase Cancer Center Assay for identifying agents that modulate epigenetic silencing, and agents identified thereby
KR102537394B1 (ko) * 2014-03-17 2023-05-30 애드베룸 바이오테크놀로지스, 인코포레이티드 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법
EP3377116A4 (en) * 2015-11-19 2019-07-10 The Trustees of The University of Pennsylvania COMPOSITIONS AND METHODS FOR CORRECTING ECG-RELATED EYE DISEASE
WO2018109011A1 (en) 2016-12-13 2018-06-21 Stichting Katholieke Universiteit Antisense oligonucleotides for the treatment of stargardt disease
AU2018223810B2 (en) 2017-02-27 2022-04-21 Case Western Reserve University Visual cycle modulators
CA3137135A1 (en) 2019-04-19 2020-10-22 University Of Massachusetts Gene therapies for stargardt disease (abca4)
EP4330409A4 (en) 2021-04-26 2025-04-09 University of Massachusetts GENE THERAPIES FOR STARGARDT'S DISEASE (ABCA4)

Also Published As

Publication number Publication date
CN114126665A (zh) 2022-03-01
WO2020214809A3 (en) 2020-11-19
WO2020214809A2 (en) 2020-10-22
US20220177539A1 (en) 2022-06-09
CA3137135A1 (en) 2020-10-22
US20260022154A1 (en) 2026-01-22
EP3955973A2 (en) 2022-02-23
US12516097B2 (en) 2026-01-06
AU2020257392A1 (en) 2021-11-18
JP2022529782A (ja) 2022-06-24
EP3955973A4 (en) 2023-01-18

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