ES2923877T3 - Vectores VAA para el tratamiento de la retinitis pigmentosa dominante - Google Patents
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- ES2923877T3 ES2923877T3 ES17760752T ES17760752T ES2923877T3 ES 2923877 T3 ES2923877 T3 ES 2923877T3 ES 17760752 T ES17760752 T ES 17760752T ES 17760752 T ES17760752 T ES 17760752T ES 2923877 T3 ES2923877 T3 ES 2923877T3
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- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- 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
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- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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| US201662302122P | 2016-03-01 | 2016-03-01 | |
| US201662398451P | 2016-09-22 | 2016-09-22 | |
| PCT/US2017/020289 WO2017151823A1 (en) | 2016-03-01 | 2017-03-01 | Aav vectors for treatment of dominant retinitis pigmentosa |
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| ES2923877T3 true ES2923877T3 (es) | 2022-10-03 |
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| IL261014B2 (en) | 2016-03-01 | 2023-10-01 | Univ Florida | Aav vectors for treatment of dominant retinitis pigmentosa |
| EP3707257A4 (en) * | 2017-11-07 | 2021-12-08 | The University of North Carolina at Chapel Hill | PROCEDURES AND COMPOSITIONS FOR CIRCULAR RNA MOLECULES |
| EP3793566A4 (en) * | 2018-05-15 | 2022-03-16 | University of Washington | Compositions and methods for reducing spliceopathy and treating rna dominance disorders |
| JP7563694B2 (ja) * | 2018-06-01 | 2024-10-08 | ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． | 優性網膜色素変性症の処置のための組成物および方法 |
| GB201817469D0 (en) * | 2018-10-26 | 2018-12-12 | Univ Oxford Innovation Ltd | Gene therapy for retinal disease |
| CN111518813B (zh) * | 2019-02-03 | 2023-04-28 | 武汉纽福斯生物科技有限公司 | 视紫红质的编码序列、其表达载体构建及其应用 |
| WO2021030745A1 (en) * | 2019-08-15 | 2021-02-18 | The Children's Hospital Of Philadelphia | Combined transgene and intron-derived mirna therapy for treatment of sca1 |
| CN113038971B (zh) * | 2020-04-21 | 2022-06-24 | 北京中因科技有限公司 | RHO-adRP基于基因编辑的方法和组合物 |
| CN111926044B (zh) * | 2020-10-12 | 2021-01-22 | 北京大学第三医院（北京大学第三临床医学院） | 结合突变rho基因的核酸分子和试剂盒 |
| EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| WO2025231487A1 (en) * | 2024-05-03 | 2025-11-06 | The Trustees Of The University Of Pennsylvania | Photoreceptor specific promotors for use in gene therapies for treatment of retinal degeneration |
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| US8551970B2 (en) | 1996-04-02 | 2013-10-08 | Optigen Patents Limited | Genetic suppression and replacement |
| US20040234999A1 (en) | 1996-04-02 | 2004-11-25 | Farrar Gwenyth Jane | Genetic suppression and replacement |
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| US20120322861A1 (en) | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| EP2140004B1 (en) * | 2007-04-12 | 2022-01-12 | The Provost, Fellows, Foundation Scholars, & the other members of Board, of the College of the Holy & Undiv. Trinity of Queen Elizabeth near Dublin | Genetic suppression and replacement |
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| ES2760263T3 (es) | 2014-03-21 | 2020-05-13 | Genzyme Corp | Tratamiento génico de la retinitis pigmentaria |
| BR112017017178A2 (pt) | 2015-02-26 | 2018-04-03 | Ionis Pharmaceuticals Inc | moduladores específicos de alelo de rodopsina de p23h |
| EP4008780A1 (en) | 2015-04-30 | 2022-06-08 | The Trustees of Columbia University in the City of New York | Gene therapy for autosomal dominant diseases |
| WO2017137493A1 (en) | 2016-02-09 | 2017-08-17 | Fondazione Telethon | Synthetic promoters and uses thereof |
| US20170348387A1 (en) | 2016-02-29 | 2017-12-07 | The Trustees Of The University Of Pennsylvania | Aav-mediated gene therapy for nphp5 lca-ciliopathy |
| IL261014B2 (en) | 2016-03-01 | 2023-10-01 | Univ Florida | Aav vectors for treatment of dominant retinitis pigmentosa |
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| IL261014B1 (en) | 2023-06-01 |
| IL261014A (en) | 2018-10-31 |
| US20190093111A1 (en) | 2019-03-28 |
| CN115896106A (zh) | 2023-04-04 |
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