CN113056561A - 用于治疗黄斑营养不良的组合物和方法 - Google Patents
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| CN111849998A (zh) * | 2020-07-29 | 2020-10-30 | 武汉纽福斯生物科技有限公司 | 编码人卵黄状黄斑病蛋白1的核酸分子及其应用 |
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| EP3870148A4 (en) * | 2018-10-25 | 2022-11-09 | Takeda Pharmaceutical Company Limited | AAV TRIPLE PLASMID SYSTEM |
| US20220089670A1 (en) * | 2018-12-28 | 2022-03-24 | University Of Rochester | Gene Therapy for BEST1 Dominant Mutations |
| US20230112568A1 (en) * | 2020-02-28 | 2023-04-13 | The Trustees Of The University Of Pennsylvania | Treating autosomal recessive bestrophinopathies and methods for evaluating same |
| EP4590841A2 (en) * | 2022-09-20 | 2025-07-30 | Aldevron, LLC | Best1 vectors and uses thereof |
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| US9163259B2 (en) * | 2012-05-04 | 2015-10-20 | Novartis Ag | Viral vectors for the treatment of retinal dystrophy |
| EP3071592B1 (en) * | 2013-11-20 | 2021-01-06 | Fondazione Telethon | Artificial dna-binding proteins and uses thereof |
| AU2014359136B2 (en) * | 2013-12-06 | 2020-06-25 | Centre Hospitalier Universitaire De Montpellier | Methods and pharmaceutical compositions for expressing a polynucleotide of interest in the retinal pigment epithelium of a subject |
| GB201322798D0 (en) * | 2013-12-20 | 2014-02-05 | Oxford Biomedica Ltd | Production system |
| RS63416B1 (sr) * | 2015-03-03 | 2022-08-31 | Fond Telethon | Sistem višestrukih vektora i njegove primene |
| WO2017083722A1 (en) * | 2015-11-11 | 2017-05-18 | Greenberg Kenneth P | Crispr compositions and methods of using the same for gene therapy |
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| US20190307900A1 (en) | 2019-10-10 |
| EP3775233A1 (en) | 2021-02-17 |
| SG11202009759SA (en) | 2020-10-29 |
| WO2019195727A1 (en) | 2019-10-10 |
| TW202003052A (zh) | 2020-01-16 |
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