JP7408228B2 - ヒト対象において非加齢性聴力障害を治療するための組成物及び方法 - Google Patents

ヒト対象において非加齢性聴力障害を治療するための組成物及び方法 Download PDF

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JP7408228B2
JP7408228B2 JP2020544478A JP2020544478A JP7408228B2 JP 7408228 B2 JP7408228 B2 JP 7408228B2 JP 2020544478 A JP2020544478 A JP 2020544478A JP 2020544478 A JP2020544478 A JP 2020544478A JP 7408228 B2 JP7408228 B2 JP 7408228B2
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amino acids
acid substitutions
amino acid
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JPWO2019165292A5 (https=
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エマニュエル ジョン サイモンズ，
ロバート ング，
エレン ライジンガー，
ハナン アル－モイエド，
ゼバスチアーン キューグラー，
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アコーオス インコーポレイテッド
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JP2020544478A 2018-02-22 2019-02-22 ヒト対象において非加齢性聴力障害を治療するための組成物及び方法 Active JP7408228B2 (ja)

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US201862634088P 2018-02-22 2018-02-22
US62/634,088 2018-02-22
PCT/US2019/019268 WO2019165292A1 (en) 2018-02-22 2019-02-22 Compositions and methods for treating non-age-associated hearing impairment in a human subject

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JP2021513863A JP2021513863A (ja) 2021-06-03
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JP2023212464A Pending JP2024023691A (ja) 2018-02-22 2023-12-15 ヒト対象において非加齢性聴力障害を治療するための組成物及び方法

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US (2) US20200390905A1 (https=)
EP (1) EP3755805A4 (https=)
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KR (2) KR20260015297A (https=)
CN (1) CN112020561A (https=)
AU (2) AU2019224121B2 (https=)
BR (1) BR112020016919A2 (https=)
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AU2018261769B2 (en) 2017-05-05 2024-03-14 The Regents Of The University Of California Compositions and methods for expressing Otoferlin
CN119410636A (zh) 2018-04-27 2025-02-11 分贝治疗公司 肌球蛋白15启动子及其用途
US11660353B2 (en) 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
WO2020148458A1 (en) * 2019-01-18 2020-07-23 Institut Pasteur Aav-mediated gene therapy restoring the otoferlin gene
AU2020218547A1 (en) 2019-02-08 2021-08-26 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
EP3921032A4 (en) 2019-02-08 2022-11-30 Decibel Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATMENT OF SENSORINEURAL HEARING LOSS USING OTOFERLIN DOUBLE VECTOR SYSTEMS
US20220396806A1 (en) 2019-07-26 2022-12-15 Akouos, Inc. Methods of treating hearing loss using a secreted target protein
CA3159549A1 (en) 2019-10-30 2021-05-06 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
JP7807380B2 (ja) * 2020-02-21 2026-01-27 アコーオス インコーポレイテッド ヒト対象において非加齢関連聴力障害を処置するための組成物および方法
AU2021247172A1 (en) 2020-04-01 2022-09-15 University Of Florida Research Foundation, Incorporated Dual AAV-MY07A vectors with improved safety for the treatment of USH1B
JP2023526047A (ja) 2020-05-13 2023-06-20 アコーオス インコーポレイテッド Ｓｌｃ２６ａ４関連難聴を治療するための組成物及び方法
MX2023007800A (es) 2020-12-29 2023-07-11 Akouos Inc Composiciones y metodos para tratar la perdida de audicion y/o la perdida de vision asociada a clrn1.
CN117295529A (zh) * 2021-02-19 2023-12-26 分贝治疗公司 使用耳畸蛋白双载体系统治疗感觉神经性听力损失的方法
CN113063944B (zh) * 2021-03-05 2023-03-24 李朴 血清gsdme在b淋巴细胞白血病诊断、疗效监测及预后评估上的用途
CN113337507B (zh) * 2021-08-05 2021-11-12 首都医科大学附属北京友谊医院 Otof 1273 (C>T)基因定点突变的耳聋小鼠模型的构建方法及其应用
WO2023036966A1 (en) * 2021-09-10 2023-03-16 Institut Pasteur Dual recombinant aav8 vector system encoding isoform 5 of otoferlin and uses thereof
CN114807224B (zh) * 2022-04-25 2023-09-29 中国人民解放军军事科学院军事医学研究院 一种可视化斑马鱼胆汁酸代谢模型的构建方法及其应用
CN114854762A (zh) * 2022-06-09 2022-08-05 上海鼎新基因科技有限公司 编码人otof的核酸序列及其应用
CN115141896A (zh) * 2022-09-04 2022-10-04 上海鼎新基因科技有限公司 一种AAV组织分布的qPCR检测方法
CN116925239B (zh) * 2023-07-17 2024-10-18 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
CN117305367A (zh) * 2023-08-21 2023-12-29 复旦大学附属眼耳鼻喉科医院 一种表达全长耳畸蛋白的双aav载体系统及其应用

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