TW202003052A - 用於治療黃斑部失養症之組成物及方法 - Google Patents
用於治療黃斑部失養症之組成物及方法 Download PDFInfo
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- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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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
- 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
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- A61K48/0083—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 administration regime
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- A—HUMAN NECESSITIES
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- A61K48/0091—Purification or manufacturing processes for gene therapy compositions
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- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
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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
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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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| PE20210918A1 (es) | 2021-05-19 |
| EA202092069A1 (ru) | 2021-03-12 |
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| US20190307900A1 (en) | 2019-10-10 |
| EP3775233A1 (en) | 2021-02-17 |
| SG11202009759SA (en) | 2020-10-29 |
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