CN111050805B - 用于治疗cngb1相关视网膜色素变性的基因疗法 - Google Patents

用于治疗cngb1相关视网膜色素变性的基因疗法

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Publication number
CN111050805B
CN111050805B CN201880031841.5A CN201880031841A CN111050805B CN 111050805 B CN111050805 B CN 111050805B CN 201880031841 A CN201880031841 A CN 201880031841A CN 111050805 B CN111050805 B CN 111050805B
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seq
polynucleotide
sequence identity
sequence
promoter
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CN111050805A (zh
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斯蒂利亚诺斯·米夏拉克基斯
马丁·比尔
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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
    • 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
    • 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
    • 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
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Plant Pathology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CN201880031841.5A 2017-03-21 2018-03-21 用于治疗cngb1相关视网膜色素变性的基因疗法 Active CN111050805B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
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

Publications (2)

Publication Number Publication Date
CN111050805A CN111050805A (zh) 2020-04-21
CN111050805B true CN111050805B (zh) 2026-04-14

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ID=62044771

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CN201880031841.5A Active CN111050805B (zh) 2017-03-21 2018-03-21 用于治疗cngb1相关视网膜色素变性的基因疗法

Country Status (10)

Country Link
US (2) US12246072B2 (https=)
EP (1) EP3600446A1 (https=)
JP (1) JP7257377B2 (https=)
CN (1) CN111050805B (https=)
AR (1) AR111188A1 (https=)
AU (1) AU2018238422B2 (https=)
CA (1) CA3056211A1 (https=)
TW (1) TW201902911A (https=)
UY (1) UY37636A (https=)
WO (1) WO2018172961A1 (https=)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL282362B2 (en) * 2018-10-15 2025-10-01 Fond Telethon Ets Intein proteins and their uses
US12605466B2 (en) * 2019-03-04 2026-04-21 University Of Florida Research Foundation, Incorporated Enhanced human opsin promoter for rod specific expression
JP7663504B2 (ja) * 2019-03-08 2025-04-16 ユニベルシテ パリ－サクレー 遺伝子置換による希少な眼疾患のための改善された治療方法
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
KR20220128607A (ko) 2019-09-18 2022-09-21 인터갈락틱 테라퓨틱스, 인크. 합성 dna 벡터 및 사용 방법
JP7703543B2 (ja) * 2020-01-17 2025-07-07 ザ ユナイテッド ステイツ オブ アメリカ アズ リプリゼンテッド バイ ザ セクレタリー、デパートメント オブ ヘルス アンド ヒューマン サービシーズ Ｃｒｘ常染色体優性網膜症の処置のための遺伝子治療
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
US12071633B2 (en) * 2020-10-13 2024-08-27 Kriya Therapeutics, Inc. Viral vector constructs for delivery of nucleic acids encoding cytokines and uses thereof for treating cancer
CN113201556B (zh) * 2021-05-14 2022-11-22 浙江大学 pSOY19-ZM2载体、其制备方法及应用
JP2024524600A (ja) * 2021-07-06 2024-07-05 インスティテュート オブ モレキュラー アンド クリニカル オフサルモロジー バーゼル 桿体光受容体における遺伝子の特異的発現のためのプロモーター
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

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015020522A1 (en) * 2013-08-05 2015-02-12 Koninklijke Nederlandse Akademie Van Wetenschappen Recombinant aav-crumbs homologue composition and methods for treating lca-8 and progressive rp

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
PL3628334T3 (pl) * 2014-03-21 2023-12-18 Genzyme Corporation Terapia genowa w retinopatii barwnikowej
EP3210632B1 (en) * 2016-02-23 2022-09-28 EyeServ GmbH Gene therapy for the treatment of a retinal degeneration disease

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015020522A1 (en) * 2013-08-05 2015-02-12 Koninklijke Nederlandse Akademie Van Wetenschappen Recombinant aav-crumbs homologue composition and methods for treating lca-8 and progressive rp

Also Published As

Publication number Publication date
CN111050805A (zh) 2020-04-21
AU2018238422B2 (en) 2025-04-03
WO2018172961A1 (en) 2018-09-27
US20200030458A1 (en) 2020-01-30
US20250262328A1 (en) 2025-08-21
CA3056211A1 (en) 2018-09-27
TW201902911A (zh) 2019-01-16
EP3600446A1 (en) 2020-02-05
US12246072B2 (en) 2025-03-11
UY37636A (es) 2018-10-31
JP7257377B2 (ja) 2023-04-13
AR111188A1 (es) 2019-06-12
AU2018238422A1 (en) 2019-10-03
JP2020511167A (ja) 2020-04-16

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