CN110892062A - 表达耳畸蛋白的组合物和方法 - Google Patents

表达耳畸蛋白的组合物和方法 Download PDF

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CN110892062A
CN110892062A CN201880029806.XA CN201880029806A CN110892062A CN 110892062 A CN110892062 A CN 110892062A CN 201880029806 A CN201880029806 A CN 201880029806A CN 110892062 A CN110892062 A CN 110892062A
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S·L·博伊
F·戴卡
W·W·豪斯维特
O·艾其尔
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University of Florida Research Foundation Inc
California State University System
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    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
    • C12N2840/445—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor for trans-splicing, e.g. polypyrimidine tract, branch point splicing

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CN201880029806.XA 2017-05-05 2018-05-04 表达耳畸蛋白的组合物和方法 Pending CN110892062A (zh)

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US201762502462P 2017-05-05 2017-05-05
US62/502,462 2017-05-05
PCT/US2018/031009 WO2018204734A1 (en) 2017-05-05 2018-05-04 Compositions and methods for expressing otoferlin

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US (2) US12359221B2 (enExample)
EP (1) EP3635100A4 (enExample)
JP (3) JP7240675B2 (enExample)
KR (2) KR102606810B1 (enExample)
CN (1) CN110892062A (enExample)
AU (2) AU2018261769B2 (enExample)
CA (1) CA3061955A1 (enExample)
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CN116925239A (zh) * 2023-07-17 2023-10-24 苏州星奥拓维生物技术有限公司 双载体系统表达Otof基因的组合物和方法
WO2025201333A1 (zh) * 2024-03-28 2025-10-02 苏州星奥拓维生物技术有限公司 递送Otof基因的双载体系统和其用途

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CN110892062A (zh) * 2017-05-05 2020-03-17 佛罗里达大学研究基金会 表达耳畸蛋白的组合物和方法
DE102018103924A1 (de) * 2018-02-21 2019-08-22 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin Gentherapeutische Behandlung von Schwerhörigkeit
AU2019224121B2 (en) * 2018-02-22 2025-08-07 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
US11660353B2 (en) 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
JP7331011B2 (ja) 2018-04-27 2023-08-22 デシベル セラピューティクス インコーポレイテッド ミオシン１５プロモーター及びその使用
WO2020093018A1 (en) * 2018-11-01 2020-05-07 University Of Florida Research Foundation, Incorporated A codon optimized otoferlin aav dual vector gene therapy
AU2020208933B2 (en) * 2019-01-18 2025-08-28 Centre National De La Recherche Scientifique (Cnrs) AAV-mediated gene therapy restoring the otoferlin gene
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CN116925239A (zh) * 2023-07-17 2023-10-24 苏州星奥拓维生物技术有限公司 双载体系统表达Otof基因的组合物和方法
WO2025016099A1 (zh) * 2023-07-17 2025-01-23 苏州星奥拓维生物技术有限公司 双载体系统表达Otof基因的组合物和方法
WO2025201333A1 (zh) * 2024-03-28 2025-10-02 苏州星奥拓维生物技术有限公司 递送Otof基因的双载体系统和其用途

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