CN119213136A - 用于将核酸递送至视网膜细胞的腺相关病毒载体 - Google Patents

用于将核酸递送至视网膜细胞的腺相关病毒载体 Download PDF

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Publication number
CN119213136A
CN119213136A CN202380040951.9A CN202380040951A CN119213136A CN 119213136 A CN119213136 A CN 119213136A CN 202380040951 A CN202380040951 A CN 202380040951A CN 119213136 A CN119213136 A CN 119213136A
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polypeptide
amino acid
seq
vector
acid sequence
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CN202380040951.9A
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Chinese (zh)
Inventor
拜恩 L·C·托马斯
L·C·托马斯拜恩
M·E·约翰逊
W·R·斯涛弗尔
B·E·奥兹图科
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University of Pittsburgh
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University of Pittsburgh
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    • 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
    • A61K48/0075—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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N2750/14145—Special targeting system for viral vectors

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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)
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  • Wood Science & Technology (AREA)
  • Virology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CN202380040951.9A 2022-03-30 2023-03-30 用于将核酸递送至视网膜细胞的腺相关病毒载体 Pending CN119213136A (zh)

Applications Claiming Priority (27)

Application Number Priority Date Filing Date Title
US202263325541P 2022-03-30 2022-03-30
US202263325542P 2022-03-30 2022-03-30
US202263325548P 2022-03-30 2022-03-30
US202263325558P 2022-03-30 2022-03-30
US202263325553P 2022-03-30 2022-03-30
US202263325544P 2022-03-30 2022-03-30
US202263325543P 2022-03-30 2022-03-30
US202263325540P 2022-03-30 2022-03-30
US202263325559P 2022-03-30 2022-03-30
US202263325562P 2022-03-30 2022-03-30
US202263325551P 2022-03-30 2022-03-30
US202263325550P 2022-03-30 2022-03-30
US202263325555P 2022-03-30 2022-03-30
US63/325,543 2022-03-30
US63/325,555 2022-03-30
US63/325,540 2022-03-30
US63/325,544 2022-03-30
US63/325,553 2022-03-30
US63/325,562 2022-03-30
US63/325,550 2022-03-30
US63/325,551 2022-03-30
US63/325,559 2022-03-30
US63/325,541 2022-03-30
US63/325,542 2022-03-30
US63/325,548 2022-03-30
US63/325,558 2022-03-30
PCT/US2023/016871 WO2023192463A2 (en) 2022-03-30 2023-03-30 Adeno-associated virus vectors for nucleic acid delivery to retinal cells

Publications (1)

Publication Number Publication Date
CN119213136A true CN119213136A (zh) 2024-12-27

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CN202380040951.9A Pending CN119213136A (zh) 2022-03-30 2023-03-30 用于将核酸递送至视网膜细胞的腺相关病毒载体

Country Status (11)

Country Link
US (1) US20250327092A1 (https=)
EP (1) EP4499848A4 (https=)
JP (1) JP2025510975A (https=)
KR (1) KR20250006360A (https=)
CN (1) CN119213136A (https=)
AU (1) AU2023243048A1 (https=)
CA (1) CA3255255A1 (https=)
IL (1) IL316014A (https=)
MX (1) MX2024011963A (https=)
TW (1) TW202405180A (https=)
WO (1) WO2023192463A2 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025072688A2 (en) * 2023-09-27 2025-04-03 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Adeno-associated virus vectors for nucleic acid delivery to retinal cells, for nucleic acid delivery across retinal regions, or for nucleic acid delivery to retinal ganglion cells and/or retinal pigment epithelium cells

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
CN110612353A (zh) * 2017-03-03 2019-12-24 加利福尼亚大学董事会 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向
CA3053154A1 (en) * 2017-06-30 2019-01-03 The Regents Of The University Of California Adeno-associated virus virions with variant capsids and methods of use thereof
AU2019227910B2 (en) * 2018-02-28 2025-07-17 The Regents Of The University Of California A modular system for gene and protein delivery based on AAV
US20220348613A1 (en) * 2019-08-28 2022-11-03 University of Pittsburgh - of the Commonwealath System of Higher Ecucation Adeno-associated viruses and methods and materials for making and using adeno-associated viruses
US20220313722A1 (en) * 2019-09-09 2022-10-06 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Methods of restoring lysosomal function of retinal pigment epithelial cells by activation of tfeb

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Publication number Publication date
WO2023192463A3 (en) 2023-12-21
MX2024011963A (es) 2024-11-08
CA3255255A1 (en) 2023-10-05
WO2023192463A2 (en) 2023-10-05
TW202405180A (zh) 2024-02-01
KR20250006360A (ko) 2025-01-10
EP4499848A4 (en) 2026-03-25
AU2023243048A1 (en) 2024-10-03
US20250327092A1 (en) 2025-10-23
IL316014A (en) 2024-11-01
JP2025510975A (ja) 2025-04-15
EP4499848A2 (en) 2025-02-05

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