CA2979229A1 - Rp2 vectors for treating x-linked retinitis pigmentosa - Google Patents
Rp2 vectors for treating x-linked retinitis pigmentosaInfo
- Publication number
- CA2979229A1 CA2979229A1 CA2979229A CA2979229A CA2979229A1 CA 2979229 A1 CA2979229 A1 CA 2979229A1 CA 2979229 A CA2979229 A CA 2979229A CA 2979229 A CA2979229 A CA 2979229A CA 2979229 A1 CA2979229 A1 CA 2979229A1
- Authority
- CA
- Canada
- Prior art keywords
- vector
- mammal
- rpgr
- retina
- protein
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
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- 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
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- 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
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
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- C12N2830/00—Vector systems having a special element relevant for transcription
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
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| PCT/US2016/022072 WO2016145345A1 (en) | 2015-03-11 | 2016-03-11 | Rp2 and rpgr vectors for treating x-linked retinitis pigmentosa |
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| HRP20241073T1 (hr) * | 2017-11-15 | 2024-11-08 | The Regents Of The University Of Michigan | Virusni vektori koji sadrže kodirajuće regije rdh12 i postupci za liječenje distrofija mrežnice |
| JOP20210054A1 (ar) * | 2018-09-21 | 2020-03-21 | Nightstarx Ltd | تركيبات وطرق لعلاج التهاب الشبكية الصباغي |
| WO2020172537A1 (en) * | 2019-02-22 | 2020-08-27 | University Of Massachusetts | Oxr1 gene therapy |
| US20220290147A1 (en) * | 2019-08-30 | 2022-09-15 | Baylor College Of Medicine | System for regulating gene expression |
| US20230142852A1 (en) * | 2020-03-04 | 2023-05-11 | University Of Massachusetts | Compositions and methods for modulating rpgr expression |
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| NZ704275A (en) * | 2012-07-11 | 2016-09-30 | Univ Florida | Aav-mediated gene therapy for rpgr x-linked retinal degeneration |
| AU2014216160B2 (en) * | 2013-02-15 | 2017-03-30 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | AAV8 retinoschisin expression vector for treating X-linked retinoschisis |
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| US9534225B2 (en) * | 2014-04-15 | 2017-01-03 | Applied Genetic Technologies Corporation | Codon optimized nucleic acid encoding a retinitis pigmentosa gtpase regulator (RPGR) |
| JP6741591B2 (ja) * | 2014-05-02 | 2020-08-19 | ジェンザイム・コーポレーション | 網膜およびｃｎｓ遺伝子治療用ａａｖベクター |
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| US20180327779A1 (en) * | 2015-03-03 | 2018-11-15 | Fondazione Telethon (IT/IT) | Multiple vector system and uses thereof |
| JP6935049B2 (ja) | 2015-03-11 | 2021-09-15 | アメリカ合衆国 | Ｘ連鎖性網膜色素変性症の治療のためのｒｐ２及びｒｐｇｒベクター |
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| JP2018509154A (ja) | 2018-04-05 |
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| JP2021019611A (ja) | 2021-02-18 |
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| US20180043035A1 (en) | 2018-02-15 |
| EP3268481B1 (en) | 2020-05-27 |
| US20200289670A1 (en) | 2020-09-17 |
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