JP7240675B2 - オトフェルリンを発現させるための組成物および方法 - Google Patents

オトフェルリンを発現させるための組成物および方法 Download PDF

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JP7240675B2
JP7240675B2 JP2019560646A JP2019560646A JP7240675B2 JP 7240675 B2 JP7240675 B2 JP 7240675B2 JP 2019560646 A JP2019560646 A JP 2019560646A JP 2019560646 A JP2019560646 A JP 2019560646A JP 7240675 B2 JP7240675 B2 JP 7240675B2
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sequence
otof
composition
aav
polynucleotide
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JP2020518268A (ja
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ボエ，サンフォード，エル．
デュカ，フランク
ハウスワース，ウイリアム，ダブリュー．
アキル，オマール
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University of California San Diego UCSD
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    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • A01K2267/03—Animal model, e.g. for test or diseases
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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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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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2019560646A 2017-05-05 2018-05-04 オトフェルリンを発現させるための組成物および方法 Active JP7240675B2 (ja)

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JP2023027073A JP7617600B2 (ja) 2017-05-05 2023-02-24 オトフェルリンを発現させるための組成物および方法
JP2024226066A JP7851563B2 (ja) 2017-05-05 2024-12-23 オトフェルリンを発現させるための組成物および方法

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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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CN (1) CN110892062A (https=)
AU (2) AU2018261769B2 (https=)
CA (1) CA3061955A1 (https=)
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JP2023070682A (ja) * 2017-05-05 2023-05-19 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． オトフェルリンを発現させるための組成物および方法
US12188041B2 (en) 2018-11-01 2025-01-07 University Of Florida Research Foundation, Incorporated Codon optimized otoferlin AAV dual vector gene therapy
US12377169B2 (en) 2020-04-01 2025-08-05 University Of Florida Research Foundation, Incorporated Dual AAV-MYO7A vectors with improved safety for the treatment of USH1B
JP7851563B2 (ja) 2017-05-05 2026-04-27 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． オトフェルリンを発現させるための組成物および方法

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DE102018103924A1 (de) * 2018-02-21 2019-08-22 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin Gentherapeutische Behandlung von Schwerhörigkeit
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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
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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
CA3159549A1 (en) 2019-10-30 2021-05-06 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
CN115066432B (zh) * 2019-10-30 2026-04-17 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组合物和方法
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
CN112125969A (zh) * 2020-09-30 2020-12-25 东南大学 生物因子rimbp2在维持内耳毛细胞特性上的应用
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
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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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
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JP2023070682A (ja) * 2017-05-05 2023-05-19 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． オトフェルリンを発現させるための組成物および方法
JP7617600B2 (ja) 2017-05-05 2025-01-20 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． オトフェルリンを発現させるための組成物および方法
JP2025063057A (ja) * 2017-05-05 2025-04-15 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． オトフェルリンを発現させるための組成物および方法
US12359221B2 (en) 2017-05-05 2025-07-15 The Regents Of The University Of California Compositions and methods for expressing otoferlin
JP7851563B2 (ja) 2017-05-05 2026-04-27 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． オトフェルリンを発現させるための組成物および方法
US12188041B2 (en) 2018-11-01 2025-01-07 University Of Florida Research Foundation, Incorporated Codon optimized otoferlin AAV dual vector gene therapy
US12377169B2 (en) 2020-04-01 2025-08-05 University Of Florida Research Foundation, Incorporated Dual AAV-MYO7A vectors with improved safety for the treatment of USH1B

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US20200157573A1 (en) 2020-05-21
CA3061955A1 (en) 2018-11-08
AU2018261769B2 (en) 2024-03-14
KR102606810B1 (ko) 2023-11-27
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AU2018261769A1 (en) 2019-11-21
JP2025063057A (ja) 2025-04-15
AU2024203084B2 (en) 2026-04-02
WO2018204734A1 (en) 2018-11-08
KR20230167138A (ko) 2023-12-07
CN110892062A (zh) 2020-03-17
JP2023070682A (ja) 2023-05-19
US12359221B2 (en) 2025-07-15
US20250367325A1 (en) 2025-12-04
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