CN114126667A - 使用糖苷水解酶的基因疗法载体向视网膜细胞的改进递送 - Google Patents

使用糖苷水解酶的基因疗法载体向视网膜细胞的改进递送 Download PDF

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CN114126667A
CN114126667A CN202080051735.0A CN202080051735A CN114126667A CN 114126667 A CN114126667 A CN 114126667A CN 202080051735 A CN202080051735 A CN 202080051735A CN 114126667 A CN114126667 A CN 114126667A
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composition
cell
gene therapy
transgene
glycoside hydrolase
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K·C·迈尔
S·利基特
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Nationwide Childrens Hospital Inc
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01018—Exo-alpha-sialidase (3.2.1.18), i.e. trans-sialidase
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    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01129—Endo-alpha-sialidase (3.2.1.129)
    • A—HUMAN NECESSITIES
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    • 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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    • 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
    • C12N2750/14171—Demonstrated in vivo effect

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
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CN202080051735.0A 2019-05-17 2020-04-15 使用糖苷水解酶的基因疗法载体向视网膜细胞的改进递送 Pending CN114126667A (zh)

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US201962849794P 2019-05-17 2019-05-17
US62/849794 2019-05-17
PCT/US2020/028207 WO2020236351A1 (en) 2019-05-17 2020-04-15 Improved delivery of gene therapy vectors to retinal cells using a glycoside hydrolase enzyme

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US (2) US20220233655A1 (https=)
EP (2) EP3969060A1 (https=)
JP (2) JP2022533645A (https=)
KR (1) KR20220009427A (https=)
CN (1) CN114126667A (https=)
AR (1) AR118696A1 (https=)
AU (2) AU2020278960A1 (https=)
CA (2) CA3141017A1 (https=)
TW (1) TW202110486A (https=)
WO (2) WO2020236351A1 (https=)

Cited By (1)

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CN115323001A (zh) * 2022-07-28 2022-11-11 深圳先进技术研究院 一种靶向视网膜的基因递送系统及其应用

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KR20260045918A (ko) * 2019-12-25 2026-04-03 루머스 리미티드 도광 광학 엘리먼트와 연관된 광학 배열체를 사용하여 눈으로부터 광을 방향 전환시키는 것에 기초한 시선 추적을 위한 광학계 및 방법
CN114516901B (zh) * 2022-03-11 2023-01-17 上海勉亦生物科技有限公司 一种神经系统高亲和性的aav载体及其应用

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Publication number Priority date Publication date Assignee Title
CN115323001A (zh) * 2022-07-28 2022-11-11 深圳先进技术研究院 一种靶向视网膜的基因递送系统及其应用
CN115323001B (zh) * 2022-07-28 2024-11-26 深圳先进技术研究院 一种靶向视网膜的基因递送系统及其应用

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EP3969060A1 (en) 2022-03-23
AR118696A1 (es) 2021-10-27
JP7707081B2 (ja) 2025-07-14
JP2022533645A (ja) 2022-07-25
AU2020278499A1 (en) 2022-01-06
KR20220009427A (ko) 2022-01-24
TW202110486A (zh) 2021-03-16
WO2020236351A1 (en) 2020-11-26
EP3969059A1 (en) 2022-03-23
US20220233655A1 (en) 2022-07-28
CA3141017A1 (en) 2020-11-26
WO2020236352A1 (en) 2020-11-26
CA3141020A1 (en) 2020-11-26
AU2020278960A1 (en) 2021-12-23
JP2022533983A (ja) 2022-07-27
US20220226507A1 (en) 2022-07-21

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