CN111886340A - 包括rdh12编码区的病毒载体和治疗视网膜营养不良的方法 - Google Patents

包括rdh12编码区的病毒载体和治疗视网膜营养不良的方法 Download PDF

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Publication number
CN111886340A
CN111886340A CN201880086649.6A CN201880086649A CN111886340A CN 111886340 A CN111886340 A CN 111886340A CN 201880086649 A CN201880086649 A CN 201880086649A CN 111886340 A CN111886340 A CN 111886340A
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China
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rdh12
human
retinal
nucleic acid
protein
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English (en)
Chinese (zh)
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D.A.汤普森
R.R.阿利
A.J.史密斯
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University of Michigan Ann Arbor
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UCL Business Ltd
University of Michigan Ann Arbor
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Priority to CN202311261068.0A priority Critical patent/CN117752822A/zh
Publication of CN111886340A publication Critical patent/CN111886340A/zh
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    • A—HUMAN NECESSITIES
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • A61K38/44—Oxidoreductases (1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/44—Oxidoreductases (1)
    • A61K38/443—Oxidoreductases (1) acting on CH-OH groups as donors, e.g. glucose oxidase, lactate dehydrogenase (1.1)
    • 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
    • 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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    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12Y101/00—Oxidoreductases acting on the CH-OH group of donors (1.1)
    • C12Y101/01—Oxidoreductases acting on the CH-OH group of donors (1.1) with NAD+ or NADP+ as acceptor (1.1.1)
    • C12Y101/01105—Retinol dehydrogenase (1.1.1.105)
    • A—HUMAN NECESSITIES
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    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • 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
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
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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
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    • 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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  • Chemical Kinetics & Catalysis (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CN201880086649.6A 2017-11-15 2018-11-15 包括rdh12编码区的病毒载体和治疗视网膜营养不良的方法 Pending CN111886340A (zh)

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CN202311261068.0A CN117752822A (zh) 2017-11-15 2018-11-15 包括rdh12编码区的病毒载体和治疗视网膜营养不良的方法

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US201762586624P 2017-11-15 2017-11-15
US62/586624 2017-11-15
PCT/US2018/061319 WO2019099696A1 (en) 2017-11-15 2018-11-15 Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies

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CN202311261068.0A Pending CN117752822A (zh) 2017-11-15 2018-11-15 包括rdh12编码区的病毒载体和治疗视网膜营养不良的方法

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US (2) US20190142909A1 (https=)
EP (2) EP4364803A3 (https=)
JP (2) JP2021502978A (https=)
KR (1) KR102704296B1 (https=)
CN (2) CN111886340A (https=)
AU (1) AU2018367606B2 (https=)
BR (1) BR112020009828A2 (https=)
DK (1) DK3710590T3 (https=)
EA (1) EA202091194A1 (https=)
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HU (1) HUE068393T2 (https=)
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MA (1) MA50914A (https=)
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SI (1) SI3710590T1 (https=)
SM (1) SMT202400321T1 (https=)
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Cited By (3)

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Publication number Priority date Publication date Assignee Title
CN115725659A (zh) * 2022-04-01 2023-03-03 首都医科大学附属北京同仁医院 一种应用视网膜原位基因编辑制作非人灵长类动物先天性黑矇模型的方法
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
CN120290554A (zh) * 2024-09-10 2025-07-11 广州市未来基因递送研究院 感光细胞特异性双向启动子及其用途

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WO2018009814A1 (en) 2016-07-08 2018-01-11 The Trustees Of The University Of Pennsylvania Methods and compositions for treatment of disorders and diseases involving rdh12
WO2022232249A1 (en) * 2021-04-27 2022-11-03 The Trustees Of Columbia University In The City Of New York Methods and compositions to regulate cholesterol efflux to prevent, treat, or cure macular degeneration
CN118453915A (zh) * 2024-04-29 2024-08-09 中国医学科学院北京协和医院 一种针对rdh12突变的基因治疗药物

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WO2011034947A2 (en) * 2009-09-15 2011-03-24 University Of Washington Reagents and methods for modulating cone photoreceptor activity
WO2016014353A1 (en) * 2014-07-24 2016-01-28 Massachusetts Eye & Ear Infirmary Rpgr gene therapy for retinitis pigmentosa

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EP2872183B1 (en) * 2012-07-11 2018-09-26 The Trustees Of The University Of Pennsylvania Aav-mediated gene therapy for rpgr x-linked retinal degeneration
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WO2011034947A2 (en) * 2009-09-15 2011-03-24 University Of Washington Reagents and methods for modulating cone photoreceptor activity
WO2016014353A1 (en) * 2014-07-24 2016-01-28 Massachusetts Eye & Ear Infirmary Rpgr gene therapy for retinitis pigmentosa

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Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115725659A (zh) * 2022-04-01 2023-03-03 首都医科大学附属北京同仁医院 一种应用视网膜原位基因编辑制作非人灵长类动物先天性黑矇模型的方法
CN120290554A (zh) * 2024-09-10 2025-07-11 广州市未来基因递送研究院 感光细胞特异性双向启动子及其用途
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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CN117752822A (zh) 2024-03-26
DK3710590T3 (da) 2024-08-12
JP7615238B2 (ja) 2025-01-16
JP2021502978A (ja) 2021-02-04
PT3710590T (pt) 2024-08-09
RS65881B1 (sr) 2024-09-30
MX2026001243A (es) 2026-03-02
ES2983990T3 (es) 2024-10-28
AU2018367606B2 (en) 2024-08-29
EA202091194A1 (ru) 2020-08-14
MA50914A (fr) 2020-09-23
EP4364803A2 (en) 2024-05-08
EP3710590A4 (en) 2021-06-23
HUE068393T2 (hu) 2024-12-28
KR102704296B1 (ko) 2024-09-05
HRP20241073T1 (hr) 2024-11-08
JP2023133434A (ja) 2023-09-22
LT3710590T (lt) 2024-09-25
US12589136B2 (en) 2026-03-31
IL274609B1 (en) 2024-01-01
CA3082586A1 (en) 2019-05-23
ZA202003416B (en) 2024-10-30
EP3710590A1 (en) 2020-09-23
PL3710590T3 (pl) 2024-10-21
IL308645B2 (en) 2026-01-01
MY204271A (en) 2024-08-20
EP3710590B1 (en) 2024-07-17
IL274609B2 (en) 2024-05-01
US20230190884A1 (en) 2023-06-22
FI3710590T3 (fi) 2024-08-06
US20190142909A1 (en) 2019-05-16
IL274609A (en) 2020-06-30
SG11202004527YA (en) 2020-06-29
SMT202400321T1 (it) 2024-11-15
IL308645B1 (en) 2025-09-01
SI3710590T1 (sl) 2024-10-30
BR112020009828A2 (pt) 2020-11-17
IL308645A (en) 2024-01-01
EP4364803A3 (en) 2024-06-05
AU2018367606A1 (en) 2020-06-25
KR20200088385A (ko) 2020-07-22
MX2020005008A (es) 2020-11-06
WO2019099696A1 (en) 2019-05-23

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