CA3025445A1 - Dual overlapping adeno-associated viral vector system for expressing abc4a - Google Patents
Dual overlapping adeno-associated viral vector system for expressing abc4a Download PDFInfo
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- CA3025445A1 CA3025445A1 CA3025445A CA3025445A CA3025445A1 CA 3025445 A1 CA3025445 A1 CA 3025445A1 CA 3025445 A CA3025445 A CA 3025445A CA 3025445 A CA3025445 A CA 3025445A CA 3025445 A1 CA3025445 A1 CA 3025445A1
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- nucleic acid
- acid sequence
- sequence
- aav vector
- abca4
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- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
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- A—HUMAN NECESSITIES
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- 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
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- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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Applications Claiming Priority (5)
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| GBGB1610448.1A GB201610448D0 (en) | 2016-06-15 | 2016-06-15 | Adeno-associated viral vector system |
| GB1610448.1 | 2016-06-15 | ||
| GB1707261.2 | 2017-05-05 | ||
| GBGB1707261.2A GB201707261D0 (en) | 2017-05-05 | 2017-05-05 | Adeno-associated viral vector system |
| PCT/GB2017/051741 WO2017216560A1 (en) | 2016-06-15 | 2017-06-14 | Dual overlapping adeno-associated viral vector system for expressing abc4a |
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| EP (1) | EP3472328A1 (https=) |
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| AU2014255665B2 (en) | 2013-04-18 | 2018-08-02 | Fondazione Telethon | Effective delivery of large genes by dual AAV vectors |
| KR102606810B1 (ko) | 2017-05-05 | 2023-11-27 | 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 | 오토펄린을 발현시키기 위한 조성물 및 방법 |
| WO2019195726A1 (en) * | 2018-04-05 | 2019-10-10 | Oxford University Innovation Limited | Compositions and methods for the treatment of stargardt disease |
| IL282362B2 (en) * | 2018-10-15 | 2025-10-01 | Fond Telethon Ets | Intein proteins and their uses |
| GB201817469D0 (en) * | 2018-10-26 | 2018-12-12 | Univ Oxford Innovation Ltd | Gene therapy for retinal disease |
| WO2020093018A1 (en) | 2018-11-01 | 2020-05-07 | University Of Florida Research Foundation, Incorporated | A codon optimized otoferlin aav dual vector gene therapy |
| 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 |
| US20220267802A1 (en) * | 2019-07-15 | 2022-08-25 | President And Fellows Of Harvard College | Methods and compositions for gene delivery |
| KR20220130093A (ko) | 2019-10-30 | 2022-09-26 | 데시벨 테라퓨틱스, 인크. | 오토펄린 듀얼 벡터 시스템을 사용한 감각신경성 난청을 치료하기 위한 조성물 및 방법 |
| CN115666658A (zh) | 2020-04-01 | 2023-01-31 | 佛罗里达州大学研究基金会 | 具有提高的治疗ush1b的安全性的双aav-myo7a载体 |
| WO2022036318A1 (en) * | 2020-08-14 | 2022-02-17 | Case Western Reserve University | Plasmid vectors and nanoparticles for treating ocular disorders |
| US20240425877A1 (en) * | 2021-05-07 | 2024-12-26 | Ucl Business Ltd | Abca4 genome editing |
| WO2023160454A1 (zh) * | 2022-02-25 | 2023-08-31 | 北京中因科技有限公司 | 表达盒组合及其应用 |
| EP4728080A1 (en) * | 2023-06-16 | 2026-04-22 | Arkasubhra Ghosh | A hybrid dual aav vector system with splice enhancer elements for expression of large genes |
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| EP2311966A3 (en) * | 2005-10-20 | 2012-09-05 | Amsterdam Molecular Therapeutics (AMT) B.V. | Improved AAV vectors produced in insect cells |
| US20090214478A1 (en) * | 2008-02-21 | 2009-08-27 | Alberto Auricchio | Method of treating ocular diseases by gene therapy |
| RU2555346C2 (ru) * | 2009-08-07 | 2015-07-10 | Трансген Са | Композиция для лечения инфекции вирусом гепатита в |
| WO2013075008A1 (en) * | 2011-11-16 | 2013-05-23 | University Of Florida Research Foundation Inc. | Aav dual vector systems for gene therapy |
| AU2014255665B2 (en) * | 2013-04-18 | 2018-08-02 | Fondazione Telethon | Effective delivery of large genes by dual AAV vectors |
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| US20190309326A1 (en) | 2019-10-10 |
| KR20190020745A (ko) | 2019-03-04 |
| WO2017216560A1 (en) | 2017-12-21 |
| MX2018015629A (es) | 2019-09-26 |
| RU2019100525A3 (https=) | 2020-07-15 |
| RU2765826C2 (ru) | 2022-02-03 |
| JP2019523648A (ja) | 2019-08-29 |
| EP3472328A1 (en) | 2019-04-24 |
| IL263523A (en) | 2019-01-31 |
| BR112018075855A2 (pt) | 2019-04-02 |
| CN109642242A (zh) | 2019-04-16 |
| SG11201811244SA (en) | 2019-01-30 |
| RU2019100525A (ru) | 2020-07-15 |
| AU2017286623A1 (en) | 2018-12-20 |
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