CN119320772A - 肌球蛋白15启动子及其用途 - Google Patents
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Classifications
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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
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4716—Muscle proteins, e.g. myosin, actin
-
- 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
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- 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
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
- 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
- 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
- A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
-
- C—CHEMISTRY; METALLURGY
- 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
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- 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
- C12N15/86—Viral vectors
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- A—HUMAN NECESSITIES
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
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- C—CHEMISTRY; METALLURGY
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- 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
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- 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
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Biophysics (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Public Health (AREA)
- Epidemiology (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
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| US202062965773P | 2020-01-24 | 2020-01-24 | |
| US62/965773 | 2020-01-24 | ||
| PCT/US2020/017292 WO2020163761A1 (en) | 2019-02-08 | 2020-02-07 | Myosin 15 promoters and uses thereof |
| CN202080026444.6A CN114072179B (zh) | 2019-02-08 | 2020-02-07 | 肌球蛋白15启动子及其用途 |
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| EP (1) | EP3920974A4 (https=) |
| JP (3) | JP7562544B2 (https=) |
| CN (3) | CN119320772A (https=) |
| AU (1) | AU2020218547A1 (https=) |
| CA (1) | CA3129422A1 (https=) |
| WO (1) | WO2020163761A1 (https=) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
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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 |
| 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 |
| 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 |
| 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 |
| CN112194717B (zh) * | 2020-09-30 | 2021-12-14 | 东南大学 | 一种促进耳蜗毛细胞再生的肽段Tr及其应用 |
| JP2025504263A (ja) | 2021-12-23 | 2025-02-10 | ユニバーシティ オブ ロチェスター | 内耳への薬剤の送達のためのｃｓｆ輸送経路 |
| JP2025503508A (ja) | 2021-12-23 | 2025-02-04 | ユニバーシティ オブ ロチェスター | 内耳への薬剤の送達のための組成物及び方法 |
| CN116836975A (zh) * | 2022-03-25 | 2023-10-03 | 上海玮美基因科技有限责任公司 | 一种耳蜗和/或前庭细胞特异性启动子及其应用 |
| WO2024250774A1 (zh) * | 2023-06-07 | 2024-12-12 | 苏州星奥拓维生物技术有限公司 | 耳蜗外毛细胞特异性启动子及其应用 |
| CN117486995A (zh) * | 2023-11-03 | 2024-02-02 | 首都医科大学附属北京胸科医院 | Kncn蛋白在结核诊断试剂中的应用 |
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| GB9720465D0 (en) | 1997-09-25 | 1997-11-26 | Oxford Biomedica Ltd | Dual-virus vectors |
| US6436392B1 (en) | 1998-05-20 | 2002-08-20 | University Of Iowa Research Foundation | Adeno-associated virus vectors |
| EP1224313A1 (en) | 1999-10-07 | 2002-07-24 | University of Iowa Research Foundation | Adeno-associated viruses and uses thereof |
| WO2001029243A1 (en) | 1999-10-15 | 2001-04-26 | Dalhousie University | Method and vector for producing and transferring trans-spliced peptides |
| US20040072154A1 (en) * | 2000-12-22 | 2004-04-15 | Morris David W. | Novel compositions and methods for cancer |
| JP2003088388A (ja) * | 2001-09-14 | 2003-03-25 | Herikkusu Kenkyusho:Kk | 新規な全長ｃＤＮＡ |
| US20060024278A1 (en) * | 2004-01-23 | 2006-02-02 | The General Hospital Corporation | Methods and products related to the production of inner ear hair cells |
| US20070161031A1 (en) * | 2005-12-16 | 2007-07-12 | The Board Of Trustees Of The Leland Stanford Junior University | Functional arrays for high throughput characterization of gene expression regulatory elements |
| US8298818B2 (en) | 2006-04-28 | 2012-10-30 | University Of Florida Research Foundation, Inc. | Self-complementary adeno-associated virus having a truncated CMV-chicken β-actin promoter |
| JP5575486B2 (ja) | 2007-01-18 | 2014-08-20 | ユニヴァーシティ オブ ミズーリー−コロンビア | 筋鞘にｎＮＯＳを回復させる合成ミニ／ミクロジストロフィン遺伝子 |
| FR2919305B1 (fr) | 2007-07-26 | 2009-09-18 | Genethon Ass Loi De 1901 | Vecteurs viraux adeno-associes pour l'expression de la dysferline. |
| EP2732819B1 (en) | 2008-02-07 | 2019-10-16 | Massachusetts Eye & Ear Infirmary | Compounds that enhance Atoh-1 expression |
| US8236557B2 (en) | 2008-05-28 | 2012-08-07 | University Of Missouri-Columbia | Hybrid-AAV vectors to deliver large gene expression cassette |
| US8962314B2 (en) | 2008-11-26 | 2015-02-24 | The Regents Of The University Of California | Lateral ventricle cell compositions and use for treating neural degenerative diseases |
| HRP20190144T1 (hr) | 2010-04-23 | 2019-03-22 | University Of Florida Research Foundation, Inc. | Pripravci raav-gvanilat ciklaze i postupci za liječenje leberove urođene amauroze-1 (lca1) |
| WO2013075008A1 (en) | 2011-11-16 | 2013-05-23 | University Of Florida Research Foundation Inc. | Aav dual vector systems for gene therapy |
| EP2802657B1 (en) * | 2012-01-12 | 2018-05-02 | Quark Pharmaceuticals, Inc. | Combination therapy for treating hearing and balance disorders |
| US20150050354A1 (en) * | 2012-04-02 | 2015-02-19 | Moderna Therapeutics, Inc. | Modified polynucleotides for the treatment of otic diseases and conditions |
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| CA2884309A1 (en) * | 2012-09-07 | 2014-03-13 | Massachusetts Eye And Ear Infirmary | Methods and compositions for regenerating hair cells and/or supporting cells from differentiated cochlear cells or differentiated utricular cells by modulation of notch and cmyc activity |
| CA2909733C (en) | 2013-04-18 | 2023-10-17 | Fondazione Telethon | Effective delivery of large genes by dual aav vectors |
| KR102234672B1 (ko) | 2013-05-21 | 2021-04-06 | 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 | 캡시드-변형된 raav3 벡터 조성물, 및 인간 간암의 유전자 요법에서의 용도 |
| US10751385B2 (en) | 2015-02-20 | 2020-08-25 | Institut Pasteur | Prevention and/or treatment of hearing loss or impairment |
| RS63416B1 (sr) | 2015-03-03 | 2022-08-31 | Fond Telethon | Sistem višestrukih vektora i njegove primene |
| SG11201804814YA (en) | 2015-12-11 | 2018-07-30 | Massachusetts Eye & Ear Infirmary | Materials and methods for delivering nucleic acids to cochlear and vestibular cells |
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| JP2019530737A (ja) | 2016-08-23 | 2019-10-24 | 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性聴力障害を治療するための組成物および方法 |
| WO2018145111A1 (en) | 2017-02-06 | 2018-08-09 | Children's Medical Center Corporation | Materials and methods for delivering nucleic acids to cochlear and vestibular cells |
| AU2018261769B2 (en) | 2017-05-05 | 2024-03-14 | The Regents Of The University Of California | Compositions and methods for expressing Otoferlin |
| DE102018103924A1 (de) | 2018-02-21 | 2019-08-22 | Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin | Gentherapeutische Behandlung von Schwerhörigkeit |
| IL325122A (en) | 2018-02-22 | 2026-02-01 | Akouos Inc | Compositions and methods for treating non-age-related hearing impairment in humans |
| 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 |
| US12188041B2 (en) | 2018-11-01 | 2025-01-07 | University Of Florida Research Foundation, Incorporated | Codon optimized otoferlin AAV dual vector gene therapy |
| CN118497276A (zh) | 2018-11-07 | 2024-08-16 | 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 |
| WO2020148458A1 (en) | 2019-01-18 | 2020-07-23 | Institut Pasteur | Aav-mediated gene therapy restoring the otoferlin gene |
| JP2022522995A (ja) | 2019-01-18 | 2022-04-21 | ボイジャー セラピューティクス インコーポレイテッド | Ａａｖ粒子を生成するための方法及びシステム |
| 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 |
| 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 |
| EP4665751A2 (en) | 2023-02-17 | 2025-12-24 | Decibel Therapeutics, Inc. | Methods and compositions for administering otoferlin dual vector systems |
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| CN114072179B (zh) | 2024-11-01 |
| JP2026020285A (ja) | 2026-02-06 |
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| US20250304637A1 (en) | 2025-10-02 |
| JP7562544B2 (ja) | 2024-10-07 |
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| US20210388045A1 (en) | 2021-12-16 |
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| US12252520B2 (en) | 2025-03-18 |
| WO2020163761A1 (en) | 2020-08-13 |
| EP3920974A4 (en) | 2022-11-30 |
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