IL302653A - Variant adeno-associated virus (aav)capsid polypeptides and gene therapeutics thereof for treatment of hearing loss - Google Patents
Variant adeno-associated virus (aav)capsid polypeptides and gene therapeutics thereof for treatment of hearing lossInfo
- Publication number
- IL302653A IL302653A IL302653A IL30265323A IL302653A IL 302653 A IL302653 A IL 302653A IL 302653 A IL302653 A IL 302653A IL 30265323 A IL30265323 A IL 30265323A IL 302653 A IL302653 A IL 302653A
- Authority
- IL
- Israel
- Prior art keywords
- gjb2
- acid sequence
- variant
- aav
- capsid polypeptide
- Prior art date
Links
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- 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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- C—CHEMISTRY; METALLURGY
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- C12N2830/00—Vector systems having a special element relevant for transcription
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- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/38—Vector systems having a special element relevant for transcription being a stuffer
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- C—CHEMISTRY; METALLURGY
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- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
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- 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
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Landscapes
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| US202163146269P | 2021-02-05 | 2021-02-05 | |
| PCT/US2021/058255 WO2022099007A1 (en) | 2020-11-06 | 2021-11-05 | Variant adeno-associated virus (aav)capsid polypeptides and gene therapeutics thereof for treatment of hearing loss |
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| CA3168055A1 (en) | 2020-02-21 | 2021-08-26 | Emmanuel John Simons | Compositions and methods for treating non-age-associated hearing impairment in a human subject |
| CN120187432A (zh) * | 2023-02-10 | 2025-06-20 | 苏州星奥拓维生物技术有限公司 | Gch1抑制剂在制备用于预防和治疗听力损伤的药物中的用途 |
| 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 |
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| WO2019200016A1 (en) * | 2018-04-10 | 2019-10-17 | President And Fellows Of Harvard College | Aav vectors encoding clarin-1 or gjb2 and uses thereof |
| CN113853437A (zh) * | 2018-11-07 | 2021-12-28 | 阿库斯股份有限公司 | 腺相关病毒载体用于在内耳中的毛细胞和支持细胞中校正基因缺陷/表达蛋白质的用途 |
| CN113924109A (zh) * | 2019-01-18 | 2022-01-11 | 巴斯德研究所 | Aav介导的基因疗法恢复耳畸蛋白基因 |
| JP7713130B2 (ja) * | 2019-09-30 | 2025-07-25 | イーライ リリー アンド カンパニー | 遺伝性難聴の処置のためのアデノ随伴ウイルス（ａａｖ）システム |
| CN111349148A (zh) * | 2020-03-13 | 2020-06-30 | 辉大(上海)生物科技有限公司 | 一种腺相关病毒载体及其用途 |
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