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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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)
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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)
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  • Virology (AREA)
  • General Engineering & Computer Science (AREA)
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  • 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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (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
US202263325548P 2022-03-30 2022-03-30
US202263325555P 2022-03-30 2022-03-30
US202263325544P 2022-03-30 2022-03-30
US202263325543P 2022-03-30 2022-03-30
US202263325558P 2022-03-30 2022-03-30
US202263325559P 2022-03-30 2022-03-30
US202263325540P 2022-03-30 2022-03-30
US202263325553P 2022-03-30 2022-03-30
US202263325551P 2022-03-30 2022-03-30
US202263325550P 2022-03-30 2022-03-30
US202263325562P 2022-03-30 2022-03-30
US202263325542P 2022-03-30 2022-03-30
US63/325,551 2022-03-30
US63/325,542 2022-03-30
US63/325,555 2022-03-30
US63/325,550 2022-03-30
US63/325,544 2022-03-30
US63/325,559 2022-03-30
US63/325,548 2022-03-30
US63/325,540 2022-03-30
US63/325,553 2022-03-30
US63/325,543 2022-03-30
US63/325,541 2022-03-30
US63/325,562 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 (OSRAM)
EP (1) EP4499848A4 (OSRAM)
JP (1) JP2025510975A (OSRAM)
KR (1) KR20250006360A (OSRAM)
CN (1) CN119213136A (OSRAM)
AU (1) AU2023243048A1 (OSRAM)
CA (1) CA3255255A1 (OSRAM)
IL (1) IL316014A (OSRAM)
MX (1) MX2024011963A (OSRAM)
TW (1) TW202405180A (OSRAM)
WO (1) WO2023192463A2 (OSRAM)

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
CN107012171B (zh) * 2011-04-22 2021-07-13 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
CN118416088A (zh) * 2017-03-03 2024-08-02 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
EP3645052A4 (en) * 2017-06-30 2021-07-28 Regents of the University of California ADENO-ASSOCIATED VIRUS VIRIONS WITH ASSOCIATED CAPSIDE VARIANTS AND METHODS OF USE
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
MX2022002652A (es) * 2019-09-09 2022-04-06 Univ Pittsburgh Commonwealth Sys Higher Education Metodos para restaurar la funcion lisosomal de celulas epiteliales de pigmento retiniano mediante activacion de tfeb.

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

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