CN117752822A - 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
CN117752822A
CN117752822A CN202311261068.0A CN202311261068A CN117752822A CN 117752822 A CN117752822 A CN 117752822A CN 202311261068 A CN202311261068 A CN 202311261068A CN 117752822 A CN117752822 A CN 117752822A
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China
Prior art keywords
rdh12
human
retinal
adeno
viral vector
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CN202311261068.0A
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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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University of Michigan Ann Arbor
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    • 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
    • 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)
    • 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
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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
    • 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
    • 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
    • A01K2227/105—Murine
    • 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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  • Immunology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • 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)
CN202311261068.0A 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 CN117752822A (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762586624P 2017-11-15 2017-11-15
US62/586624 2017-11-15
CN201880086649.6A CN111886340A (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
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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CN117752822A true CN117752822A (zh) 2024-03-26

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CN201880086649.6A Pending CN111886340A (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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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN119746103A (zh) * 2024-04-29 2025-04-04 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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KR102526506B1 (ko) 2016-07-08 2023-05-03 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Rdh12가 연루된 장애 및 질환의 치료를 위한 방법 및 조성물
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
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
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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US20120172419A1 (en) * 2009-09-15 2012-07-05 Medical College Of Wisconsin Research Foundation Inc. Reagents and methods for modulating cone photoreceptor activity
SG10201800541SA (en) * 2010-04-23 2018-03-28 Univ Florida Raav-guanylate cyclase compositions and methods for treating leber's congenital amaurosis-1 (lca1)
WO2012167109A2 (en) * 2011-06-03 2012-12-06 Massachusetts Eye & Ear Infirmary Rpgrip1 gene therapy for leber congenital amaurosis
JP6199965B2 (ja) * 2012-07-11 2017-09-20 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Ｒｐｇｒｘ連鎖性網膜変性のａａｖ媒介型遺伝子治療
AU2015252797C1 (en) * 2014-05-02 2021-04-22 Genzyme Corporation AAV vectors for retinal and CNS gene therapy
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AU2016228751B2 (en) * 2015-03-11 2022-01-13 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Rp2 vectors for treating x-linked retinitis pigmentosa
WO2016176690A2 (en) * 2015-04-30 2016-11-03 The Trustees Of Columbia University In The City Of New York Gene therapy for autosomal dominant diseases
KR102526506B1 (ko) 2016-07-08 2023-05-03 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Rdh12가 연루된 장애 및 질환의 치료를 위한 방법 및 조성물

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN119746103A (zh) * 2024-04-29 2025-04-04 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
CN119746103B (zh) * 2024-04-29 2025-10-28 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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MA50914A (fr) 2020-09-23
AU2018367606B2 (en) 2024-08-29
HUE068393T2 (hu) 2024-12-28
KR102704296B1 (ko) 2024-09-05
JP2021502978A (ja) 2021-02-04
ES2983990T3 (es) 2024-10-28
BR112020009828A2 (pt) 2020-11-17
IL274609A (en) 2020-06-30
CA3082586A1 (en) 2019-05-23
ZA202003416B (en) 2024-10-30
EP3710590A1 (en) 2020-09-23
US20190142909A1 (en) 2019-05-16
IL274609B1 (en) 2024-01-01
EA202091194A1 (ru) 2020-08-14
KR20200088385A (ko) 2020-07-22
MX2020005008A (es) 2020-11-06
JP2023133434A (ja) 2023-09-22
IL308645B1 (en) 2025-09-01
SMT202400321T1 (it) 2024-11-15
SG11202004527YA (en) 2020-06-29
US20230190884A1 (en) 2023-06-22
IL274609B2 (en) 2024-05-01
IL308645A (en) 2024-01-01
MY204271A (en) 2024-08-20
CN111886340A (zh) 2020-11-03
RS65881B1 (sr) 2024-09-30
SI3710590T1 (sl) 2024-10-30
PL3710590T3 (pl) 2024-10-21
EP3710590B1 (en) 2024-07-17
FI3710590T3 (fi) 2024-08-06
DK3710590T3 (da) 2024-08-12
AU2018367606A1 (en) 2020-06-25
EP4364803A2 (en) 2024-05-08
JP7615238B2 (ja) 2025-01-16
EP3710590A4 (en) 2021-06-23
WO2019099696A1 (en) 2019-05-23
HRP20241073T1 (hr) 2024-11-08
EP4364803A3 (en) 2024-06-05
PH12020551319A1 (en) 2021-09-01
PT3710590T (pt) 2024-08-09

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