CN111819286B - 通过衣壳修饰增加组织特异性的基因递送 - Google Patents

通过衣壳修饰增加组织特异性的基因递送 Download PDF

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CN111819286B
CN111819286B CN201980018151.0A CN201980018151A CN111819286B CN 111819286 B CN111819286 B CN 111819286B CN 201980018151 A CN201980018151 A CN 201980018151A CN 111819286 B CN111819286 B CN 111819286B
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CN111819286A (zh
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斯科特·艾伦·卢伊勒
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Nationwide Childrens Hospital Inc
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    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C12N2310/00—Structure or type of the nucleic acid
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    • 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
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    • 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)
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  • Wood Science & Technology (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Mycology (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)
CN201980018151.0A 2018-03-16 2019-03-14 通过衣壳修饰增加组织特异性的基因递送 Active CN111819286B (zh)

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US201862644317P 2018-03-16 2018-03-16
US62/644,317 2018-03-16
PCT/US2019/022353 WO2019178412A1 (en) 2018-03-16 2019-03-14 Increasing tissue specific gene delivery by capsid modification

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US (2) US12385062B2 (https=)
EP (1) EP3765624A4 (https=)
JP (3) JP7304878B2 (https=)
KR (3) KR20250111395A (https=)
CN (2) CN111819286B (https=)
AU (2) AU2019234918C1 (https=)
BR (1) BR112020018354A2 (https=)
CA (3) CA3293599A1 (https=)
IL (2) IL317905A (https=)
SG (1) SG11202008687XA (https=)
WO (1) WO2019178412A1 (https=)

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ES2865487T3 (es) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
EP3765624A4 (en) * 2018-03-16 2022-05-25 Research Institute at Nationwide Children's Hospital Increasing tissue specific gene delivery by capsid modification
EP3774852A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
AU2019247748A1 (en) 2018-04-03 2020-10-08 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
US12611436B2 (en) 2019-10-17 2026-04-28 Sarepta Therapeutics, Inc. AAV transfer cassette
JP2022551739A (ja) 2019-10-17 2022-12-13 ストライドバイオ，インコーポレイテッド ニーマン・ピック病ｃ型の治療のためのアデノ随伴ウイルスベクター
AU2021328475A1 (en) 2020-08-19 2023-03-16 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of Rett syndrome
US20240299586A1 (en) * 2021-05-06 2024-09-12 The Regents Of The University Of California Novel aav serotypes derived from a library screen
WO2023196862A1 (en) 2022-04-06 2023-10-12 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
US20240384271A1 (en) * 2023-04-05 2024-11-21 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy

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CN102174574A (zh) * 2003-09-30 2011-09-07 宾夕法尼亚州立大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
CN104487579A (zh) * 2012-04-18 2015-04-01 费城儿童医院 使用aav衣壳变异体的高度有效的基因转移的组合物和方法
CN105579465A (zh) * 2013-07-22 2016-05-11 费城儿童医院 用于基因转移到细胞、器官和组织中的变异aav和组合物、方法及用途
WO2017058892A2 (en) * 2015-09-28 2017-04-06 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
CN107073051A (zh) * 2014-10-21 2017-08-18 马萨诸塞大学 重组aav变体及其用途

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CN104487579A (zh) * 2012-04-18 2015-04-01 费城儿童医院 使用aav衣壳变异体的高度有效的基因转移的组合物和方法
CN105579465A (zh) * 2013-07-22 2016-05-11 费城儿童医院 用于基因转移到细胞、器官和组织中的变异aav和组合物、方法及用途
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IL277270B1 (en) 2025-01-01
JP2023115149A (ja) 2023-08-18
US12385062B2 (en) 2025-08-12
KR20250111393A (ko) 2025-07-22
CN111819286A (zh) 2020-10-23
KR20200131843A (ko) 2020-11-24
IL277270B2 (en) 2025-05-01
EP3765624A4 (en) 2022-05-25
AU2025204275A1 (en) 2025-07-03
EP3765624A1 (en) 2021-01-20
BR112020018354A2 (pt) 2020-12-29
IL317905A (en) 2025-02-01
CN118373887A (zh) 2024-07-23
KR20250111395A (ko) 2025-07-22
CA3092353A1 (en) 2019-09-19
IL277270A (en) 2020-10-29
RU2020131998A (ru) 2022-04-18
JP2024144521A (ja) 2024-10-11
JP2021515572A (ja) 2021-06-24
JP7304878B2 (ja) 2023-07-07
SG11202008687XA (en) 2020-10-29
AU2019234918B2 (en) 2025-08-14
US20210087584A1 (en) 2021-03-25
AU2019234918A1 (en) 2020-09-17
AU2019234918C1 (en) 2026-03-05
CA3293598A1 (en) 2026-03-02
US20250346924A1 (en) 2025-11-13
KR102834868B1 (ko) 2025-07-17
CA3293599A1 (en) 2026-03-02
WO2019178412A1 (en) 2019-09-19

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