TW202304954A - 用於肌肉萎縮症之基因療法的aavrh74載體 - Google Patents

用於肌肉萎縮症之基因療法的aavrh74載體 Download PDF

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TW202304954A
TW202304954A TW111115475A TW111115475A TW202304954A TW 202304954 A TW202304954 A TW 202304954A TW 111115475 A TW111115475 A TW 111115475A TW 111115475 A TW111115475 A TW 111115475A TW 202304954 A TW202304954 A TW 202304954A
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阿倫 斯里瓦斯塔瓦
科云 卿
巴里 約翰 伯恩
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美商佛羅里達大學研究基金會公司
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    • A—HUMAN NECESSITIES
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • 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
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    • C12N15/86—Viral vectors
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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
    • C12N2750/14145—Special targeting system for viral vectors

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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TW111115475A 2021-04-23 2022-04-22 用於肌肉萎縮症之基因療法的aavrh74載體 TW202304954A (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202163179097P 2021-04-23 2021-04-23
US63/179,097 2021-04-23
US202263327410P 2022-04-05 2022-04-05
US63/327,410 2022-04-05

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TW202304954A true TW202304954A (zh) 2023-02-01

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US (2) US20240181083A1 (https=)
EP (1) EP4326857A2 (https=)
JP (1) JP2024514962A (https=)
KR (1) KR20240000542A (https=)
AU (1) AU2022262407A1 (https=)
BR (1) BR112023021495A2 (https=)
CA (1) CA3217649A1 (https=)
CL (2) CL2023003102A1 (https=)
CO (1) CO2023015911A2 (https=)
IL (1) IL307881A (https=)
MX (1) MX2023012509A (https=)
TW (1) TW202304954A (https=)
WO (1) WO2022226289A2 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2714007T3 (es) 2007-04-09 2019-05-24 Univ Florida Composiciones de vectores rAAV que tienen proteínas de la cápside modificadas en tirosina y métodos para su uso
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
US12358954B2 (en) 2012-05-15 2025-07-15 University Of Florida Research Foundation, Incorporated Capsid-modified rAAV vector compositions and methods therefor
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
WO2024184781A1 (en) * 2023-03-03 2024-09-12 Universitat Heidelberg Engineered aav capsids

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NZ701693A (en) * 2012-04-18 2017-02-24 The Children’S Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using aav capsid variants
US10900053B2 (en) 2014-11-21 2021-01-26 University Of Florida Research Foundation, Incorporated Genome-modified recombinant adeno-associated virus vectors
WO2021030764A1 (en) * 2019-08-14 2021-02-18 University Of Florida Research Foundation, Incorporated Aav capsid variants for gene therapy

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BR112023021495A2 (pt) 2023-12-19
IL307881A (en) 2023-12-01
US20220347317A1 (en) 2022-11-03
AU2022262407A1 (en) 2023-10-26
MX2023012509A (es) 2024-01-03
WO2022226289A3 (en) 2023-03-02
CO2023015911A2 (es) 2023-11-30
EP4326857A2 (en) 2024-02-28
US20240181083A1 (en) 2024-06-06
AU2022262407A9 (en) 2023-11-09
CL2024002556A1 (es) 2025-01-10
JP2024514962A (ja) 2024-04-03
CL2023003102A1 (es) 2024-07-12
WO2022226289A2 (en) 2022-10-27
CA3217649A1 (en) 2022-10-27
KR20240000542A (ko) 2024-01-02

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