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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    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
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    • C12N15/09—Recombinant DNA-technology
    • 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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • 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
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • 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
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
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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
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/40—Systems of functionally co-operating vectors
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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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  • Wood Science & Technology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (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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JP2023027073A Active 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 Active 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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EP (1) EP3635100A4 (https=)
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KR (2) KR102606810B1 (https=)
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

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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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EP4295913A3 (en) * 2019-01-18 2024-03-06 Institut Pasteur Aav-mediated gene therapy restoring the otoferlin gene
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KR20220130093A (ko) * 2019-10-30 2022-09-26 데시벨 테라퓨틱스, 인크. 오토펄린 듀얼 벡터 시스템을 사용한 감각신경성 난청을 치료하기 위한 조성물 및 방법
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
KR20220157944A (ko) 2020-02-21 2022-11-29 아카우오스, 인크. 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법
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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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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EP3635100A4 (en) 2021-03-24
CN110892062A (zh) 2020-03-17
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US20200157573A1 (en) 2020-05-21
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