JP7560447B2 - Ａａｖトリプルプラスミドシステム - Google Patents

Ａａｖトリプルプラスミドシステム Download PDF

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JP7560447B2
JP7560447B2 JP2021516750A JP2021516750A JP7560447B2 JP 7560447 B2 JP7560447 B2 JP 7560447B2 JP 2021516750 A JP2021516750 A JP 2021516750A JP 2021516750 A JP2021516750 A JP 2021516750A JP 7560447 B2 JP7560447 B2 JP 7560447B2
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plasmid
nucleic acid
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JP2022511348A (ja
JP2022511348A5 (enExample
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チョイ，ヴィヴィアン
リー，シン
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バクスアルタ インコーポレイテッド
バクスアルタ ゲーエムベーハー
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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
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    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • 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
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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
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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
    • C12N2750/14141—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
    • C12N2750/14151—Methods of production or purification of viral material
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/50—Vectors for producing vectors

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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
JP2021516750A 2018-10-25 2019-10-24 Ａａｖトリプルプラスミドシステム Active JP7560447B2 (ja)

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US201862750603P 2018-10-25 2018-10-25
US62/750,603 2018-10-25
PCT/US2019/057916 WO2020086881A1 (en) 2018-10-25 2019-10-24 Aav triple-plasmid system

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JP2022511348A JP2022511348A (ja) 2022-01-31
JP2022511348A5 JP2022511348A5 (enExample) 2022-10-19
JP7560447B2 true JP7560447B2 (ja) 2024-10-02

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EP (1) EP3870148A4 (enExample)
JP (2) JP7560447B2 (enExample)
KR (1) KR20210086645A (enExample)
CN (1) CN112888426A (enExample)
AU (1) AU2019363593A1 (enExample)
CA (1) CA3112883A1 (enExample)
IL (1) IL281909A (enExample)
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US20230241249A1 (en) * 2020-07-07 2023-08-03 The Board Of Regents Of The University Of Texas System Adeno-associated virus vector for dwarf open reading frame
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WO2022087321A1 (en) * 2020-10-21 2022-04-28 Duke University Dual aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy
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CN117897167A (zh) * 2021-05-13 2024-04-16 福奇生物制品公司 腺病毒辅助质粒
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WO2023102736A1 (zh) * 2021-12-07 2023-06-15 中国科学院深圳先进技术研究院 一种重组腺相关病毒包装质粒、重组腺相关病毒及其应用
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JP2022511348A (ja) 2022-01-31
WO2020086881A1 (en) 2020-04-30
EP3870148A1 (en) 2021-09-01
TW202029957A (zh) 2020-08-16
IL281909A (en) 2021-05-31
AU2019363593A1 (en) 2021-04-29
CN112888426A (zh) 2021-06-01
KR20210086645A (ko) 2021-07-08
JP2025000711A (ja) 2025-01-07
EP3870148A4 (en) 2022-11-09
US20210275614A1 (en) 2021-09-09
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