TW201902911A - 用於治療ｃｎｇｂ１關聯之視網膜色素病變的基因療法 - Google Patents
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Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- 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
-
- 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
- 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
-
- 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
- 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
-
- 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
Landscapes
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- Chemical & Material Sciences (AREA)
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- Plant Pathology (AREA)
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- Epidemiology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
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| US201762474409P | 2017-03-21 | 2017-03-21 | |
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| TW201902911A true TW201902911A (zh) | 2019-01-16 |
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| CA3116606A1 (en) * | 2018-10-15 | 2020-04-23 | Fondazione Telethon | Intein proteins and uses thereof |
| US12605466B2 (en) * | 2019-03-04 | 2026-04-21 | 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 |
| WO2021055760A1 (en) | 2019-09-18 | 2021-03-25 | Intergalactic Therapeutics, Inc. | Synthetic dna vectors and methods of use |
| 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 |
| WO2021188761A1 (en) * | 2020-03-18 | 2021-09-23 | 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 |
| CN117480256A (zh) * | 2021-03-19 | 2024-01-30 | 星际治疗有限公司 | 治疗剂的眼递送 |
| 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 |
| WO2026087373A1 (en) * | 2024-10-21 | 2026-04-30 | MeiraGTx Ocular UK Limited | Promoters for driving gene expression in rod photoreceptors and uses thereof |
| 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 |
| CN122303228A (zh) * | 2024-12-30 | 2026-06-30 | 广州市未来基因递送研究院 | 启动子及其应用 |
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| US8257969B2 (en) * | 2007-04-12 | 2012-09-04 | The Provost Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth | Genetic suppression and replacement |
| CN105980569A (zh) * | 2013-08-05 | 2016-09-28 | 莱顿大学医学中心附属莱顿教学医院 | 重组aav-crumbs同源物组合物及治疗lca-8和进行性rp的方法 |
| PT3628334T (pt) * | 2014-03-21 | 2023-09-26 | Genzyme Corp | Terapia genética para retinopatia pigmentar |
| 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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- 2018-03-21 JP JP2020501858A patent/JP7257377B2/ja active Active
- 2018-03-21 AR ARP180100661A patent/AR111188A1/es unknown
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2025
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| US20250262328A1 (en) | 2025-08-21 |
| WO2018172961A1 (en) | 2018-09-27 |
| CA3056211A1 (en) | 2018-09-27 |
| UY37636A (es) | 2018-10-31 |
| JP2020511167A (ja) | 2020-04-16 |
| US12246072B2 (en) | 2025-03-11 |
| EP3600446A1 (en) | 2020-02-05 |
| AU2018238422A1 (en) | 2019-10-03 |
| AU2018238422B2 (en) | 2025-04-03 |
| AR111188A1 (es) | 2019-06-12 |
| US20200030458A1 (en) | 2020-01-30 |
| JP7257377B2 (ja) | 2023-04-13 |
| CN111050805A (zh) | 2020-04-21 |
| CN111050805B (zh) | 2026-04-14 |
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