CN111050805A - 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 Download PDF

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CN111050805A
CN111050805A CN201880031841.5A CN201880031841A CN111050805A CN 111050805 A CN111050805 A CN 111050805A CN 201880031841 A CN201880031841 A CN 201880031841A CN 111050805 A CN111050805 A CN 111050805A
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polynucleotide
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promoter
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斯蒂利亚诺斯·米夏拉克基斯
马丁·比尔
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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
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A—HUMAN NECESSITIES
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    • 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • 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
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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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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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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 Pending CN111050805A (zh)

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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CN111050805A true CN111050805A (zh) 2020-04-21

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CN201880031841.5A Pending CN111050805A (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

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US (2) US12246072B2 (enExample)
EP (1) EP3600446A1 (enExample)
JP (1) JP7257377B2 (enExample)
CN (1) CN111050805A (enExample)
AR (1) AR111188A1 (enExample)
AU (1) AU2018238422B2 (enExample)
CA (1) CA3056211A1 (enExample)
TW (1) TW201902911A (enExample)
UY (1) UY37636A (enExample)
WO (1) WO2018172961A1 (enExample)

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
CN113201556A (zh) * 2021-05-14 2021-08-03 浙江大学 pSOY19-ZM2载体、其制备方法及应用
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
CN119432856A (zh) * 2024-12-30 2025-02-14 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

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113348249B (zh) * 2018-10-15 2025-09-23 特莱索恩基金会 内蛋白及其用途
WO2020180928A1 (en) * 2019-03-04 2020-09-10 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
CN115066255A (zh) 2019-09-18 2022-09-16 星际治疗有限公司 合成的dna载体和使用方法
CA3165922A1 (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
WO2022081776A1 (en) * 2020-10-13 2022-04-21 Kriya Therapeutics, Inc. Viral vector constructs for delivery of nucleic acids encoding cytokines and uses thereof for treating cancer
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

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US20100190841A1 (en) * 2007-04-12 2010-07-29 The Provost, Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabe Genetic suppression and replacement
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
CN106456660A (zh) * 2014-03-21 2017-02-22 建新公司 用于色素性视网膜炎的基因疗法

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EP3210632B1 (en) * 2016-02-23 2022-09-28 EyeServ GmbH Gene therapy for the treatment of a retinal degeneration disease

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US20100190841A1 (en) * 2007-04-12 2010-07-29 The Provost, Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabe Genetic suppression and replacement
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
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
CN106456660A (zh) * 2014-03-21 2017-02-22 建新公司 用于色素性视网膜炎的基因疗法

Non-Patent Citations (3)

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DEVCHAND PAUL等: "Construction of a recombinant adeno-associated virus (rAAV) vector expressing murine interleukin-12 (IL-12)", CANCER GENE THERAPY, vol. 7, no. 2, 1 February 2000 (2000-02-01), pages 308 - 315, XP037756445, DOI: 10.1038/sj.cgt.7700105 *
LEIGH A MAY BSC(HONS)等: "In vitro comparison studies of truncated rhodopsin promoter fragments from various species in human cell lines", CLINICAL AND EXPERIMENTAL OPHTHALMOLOGY, no. 31, 1 October 2003 (2003-10-01), pages 445 - 450, XP002477126, DOI: 10.1046/j.1442-9071.2003.00694.x *
SUSANNE KOCH等: "Gene therapy restores vision and delays degeneration in the CNGB12/2 mouse model of retinitis pigmentosa", HUMAN MOLECULAR GENETICS, 16 July 2012 (2012-07-16), pages 4486 - 4496 *

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
CN113201556A (zh) * 2021-05-14 2021-08-03 浙江大学 pSOY19-ZM2载体、其制备方法及应用
CN113201556B (zh) * 2021-05-14 2022-11-22 浙江大学 pSOY19-ZM2载体、其制备方法及应用
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
CN119432856A (zh) * 2024-12-30 2025-02-14 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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AU2018238422A1 (en) 2019-10-03
US20200030458A1 (en) 2020-01-30
WO2018172961A1 (en) 2018-09-27
JP7257377B2 (ja) 2023-04-13
EP3600446A1 (en) 2020-02-05
AR111188A1 (es) 2019-06-12
UY37636A (es) 2018-10-31
CA3056211A1 (en) 2018-09-27
US12246072B2 (en) 2025-03-11
AU2018238422B2 (en) 2025-04-03
US20250262328A1 (en) 2025-08-21
TW201902911A (zh) 2019-01-16
JP2020511167A (ja) 2020-04-16

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