CA3056211A1 - Gene therapy for the treatment of cngb1-linked retinitis pigmentosa - Google Patents
Gene therapy for the treatment of cngb1-linked retinitis pigmentosa Download PDFInfo
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- CA3056211A1 CA3056211A1 CA3056211A CA3056211A CA3056211A1 CA 3056211 A1 CA3056211 A1 CA 3056211A1 CA 3056211 A CA3056211 A CA 3056211A CA 3056211 A CA3056211 A CA 3056211A CA 3056211 A1 CA3056211 A1 CA 3056211A1
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- 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
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
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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
- 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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- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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Applications Claiming Priority (3)
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| US201762474409P | 2017-03-21 | 2017-03-21 | |
| US62/474,409 | 2017-03-21 | ||
| PCT/IB2018/051905 WO2018172961A1 (en) | 2017-03-21 | 2018-03-21 | Gene therapy for the treatment of cngb1-linked retinitis pigmentosa |
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| CA3056211A1 true CA3056211A1 (en) | 2018-09-27 |
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| US20220175969A1 (en) * | 2019-03-04 | 2022-06-09 | University Of Florida Research Foundation, Incorporated | Enhanced human opsin promoter for rod specific expression |
| WO2020182722A1 (en) * | 2019-03-08 | 2020-09-17 | Universite Paris-Saclay | Improved therapeutic method for rare ocular diseases by gene replacement |
| CN110241202A (zh) * | 2019-06-25 | 2019-09-17 | 复旦大学附属眼耳鼻喉科医院 | 视网膜色素变性突变位点及其应用 |
| JP2022549138A (ja) | 2019-09-18 | 2022-11-24 | インターガラクティック セラピューティクス インコーポレイテッド | 合成ｄｎａベクターおよびその使用法 |
| WO2021146625A1 (en) * | 2020-01-17 | 2021-07-22 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Gene therapy for treatment of crx-autosomal dominant retinopathies |
| US20230174598A1 (en) * | 2020-03-18 | 2023-06-08 | University Of Florida Research Foundation, Incorporated | Modified arrestin-1 to enhance photoreceptor survival in retinal disease |
| 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 |
| JP2023548746A (ja) * | 2020-10-13 | 2023-11-21 | クリヤ セラピューティクス インコーポレイテッド | サイトカインをコードする核酸を送達するためのウイルスベクターコンストラクトおよびがんを処置するためのその使用 |
| CN113201556B (zh) * | 2021-05-14 | 2022-11-22 | 浙江大学 | pSOY19-ZM2载体、其制备方法及应用 |
| WO2023280388A1 (en) * | 2021-07-06 | 2023-01-12 | Institute Of Molecular And Clinical Ophthalmology Basel | Promoter for specific expression of genes in rod photoreceptors |
| CN115820653A (zh) * | 2021-09-17 | 2023-03-21 | 深圳华大生命科学研究院 | 基因突变体及其应用 |
| CN119432856B (zh) * | 2024-12-30 | 2025-08-12 | 云舟生物科技(广州)股份有限公司 | 核酸分子、病毒颗粒及其制备方法和应用 |
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| 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 |
| US11246947B2 (en) | 2013-08-05 | 2022-02-15 | Academisch Ziekenhuis Leiden H.O.D. N. Leids Universitair Medisch Centrum | Recombinant AAV-crumbs homologue composition and methods for treating LCA-8 and progressive RP |
| ES2760263T3 (es) * | 2014-03-21 | 2020-05-13 | Genzyme Corp | Tratamiento génico de la retinitis pigmentaria |
| EP3210632B1 (en) * | 2016-02-23 | 2022-09-28 | EyeServ GmbH | Gene therapy for the treatment of a retinal degeneration disease |
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2018
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2025
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| US20250262328A1 (en) | 2025-08-21 |
| UY37636A (es) | 2018-10-31 |
| JP2020511167A (ja) | 2020-04-16 |
| US12246072B2 (en) | 2025-03-11 |
| JP7257377B2 (ja) | 2023-04-13 |
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| EP3600446A1 (en) | 2020-02-05 |
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| AR111188A1 (es) | 2019-06-12 |
| US20200030458A1 (en) | 2020-01-30 |
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