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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- 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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- C07—ORGANIC CHEMISTRY
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- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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| 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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| US12359221B2 (en) | 2017-05-05 | 2025-07-15 | The Regents Of The University Of California | Compositions and methods for expressing otoferlin |
| US20210147870A1 (en) * | 2018-04-05 | 2021-05-20 | Nightstarx Limited | Compositions and methods for the treatment of stargardt disease |
| CA3116606A1 (en) * | 2018-10-15 | 2020-04-23 | Fondazione Telethon | Intein proteins and uses thereof |
| 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 |
| WO2021015997A1 (en) * | 2019-07-15 | 2021-01-28 | President And Fellows Of Harvard College | Methods and compositions for gene delivery |
| TWI896570B (zh) | 2019-10-30 | 2025-09-11 | 美商迪賽博治療公司 | 使用耳畸蛋白雙載體系統治療感覺神經性聽力損失之組合物及方法 |
| IL295741A (en) * | 2020-04-01 | 2022-10-01 | Univ Florida | Dual aav-myo7a vectors with improved safety for the treatment of ush1b |
| US20230338580A1 (en) * | 2020-08-14 | 2023-10-26 | 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 |
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| US20090214478A1 (en) * | 2008-02-21 | 2009-08-27 | Alberto Auricchio | Method of treating ocular diseases by gene therapy |
| CA2770075C (en) * | 2009-08-07 | 2021-08-24 | Perrine Martin | Composition for treating hbv infection |
| WO2013075008A1 (en) * | 2011-11-16 | 2013-05-23 | University Of Florida Research Foundation Inc. | Aav dual vector systems for gene therapy |
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| IL263523A (en) | 2019-01-31 |
| WO2017216560A1 (en) | 2017-12-21 |
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| JP2019523648A (ja) | 2019-08-29 |
| RU2019100525A (ru) | 2020-07-15 |
| AU2017286623A1 (en) | 2018-12-20 |
| EP3472328A1 (en) | 2019-04-24 |
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| SG11201811244SA (en) | 2019-01-30 |
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