CN115379834A - 用于nmnat1相关视网膜变性的基因疗法 - Google Patents

用于nmnat1相关视网膜变性的基因疗法 Download PDF

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Publication number
CN115379834A
CN115379834A CN202180027825.0A CN202180027825A CN115379834A CN 115379834 A CN115379834 A CN 115379834A CN 202180027825 A CN202180027825 A CN 202180027825A CN 115379834 A CN115379834 A CN 115379834A
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China
Prior art keywords
nmnat1
vector
promoter
retina
aav2
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Pending
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CN202180027825.0A
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English (en)
Chinese (zh)
Inventor
E.A.皮尔斯
L.H.范登伯格
S.格林沃尔德
E.布朗
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University of California San Diego UCSD
Massachusetts Eye and Ear
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Massachusetts Eye and Ear Infirmary
University of California San Diego UCSD
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Publication of CN115379834A publication Critical patent/CN115379834A/zh
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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
    • A—HUMAN NECESSITIES
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    • 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
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    • 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
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • 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
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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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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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/07—Nucleotidyltransferases (2.7.7)
    • C12Y207/07001—Nicotinamide-nucleotide adenylyltransferase (2.7.7.1)

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  • Ophthalmology & Optometry (AREA)
  • Biomedical Technology (AREA)
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CN202180027825.0A 2020-03-11 2021-03-11 用于nmnat1相关视网膜变性的基因疗法 Pending CN115379834A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062988260P 2020-03-11 2020-03-11
US62/988,260 2020-03-11
PCT/US2021/021936 WO2021183779A1 (en) 2020-03-11 2021-03-11 Gene therapy for nmnat1-associated retinal degeneration

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CN115379834A true CN115379834A (zh) 2022-11-22

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US (2) US12257320B2 (https=)
EP (1) EP4117644A4 (https=)
JP (1) JP2023517929A (https=)
KR (1) KR20220152550A (https=)
CN (1) CN115379834A (https=)
AU (1) AU2021236233A1 (https=)
BR (1) BR112022017970A2 (https=)
CA (1) CA3174781A1 (https=)
IL (1) IL296262A (https=)
MX (1) MX2022011177A (https=)
WO (1) WO2021183779A1 (https=)

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1964627A (zh) * 2004-06-04 2007-05-16 华盛顿大学 治疗神经病变的方法和组合物
US20150259395A1 (en) * 2014-03-17 2015-09-17 Avalanche Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
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
CN106661591A (zh) * 2014-05-02 2017-05-10 建新公司 用于视网膜及cns基因疗法的aav载体
CN107206105A (zh) * 2014-07-24 2017-09-26 麻省眼耳科医院 色素性视网膜炎的rpgr基因疗法
US20180344719A1 (en) * 2015-10-23 2018-12-06 The Jackson Laboratory Treatment and prevention of ocular neurodegenerative disorder

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL3265568T3 (pl) * 2015-03-06 2020-11-30 Massachusetts Eye & Ear Infirmary Terapie augmentacji genów w przypadku wrodzonego zwyrodnienia siatkówki spowodowanego mutacjami genu prpf31
US11260081B2 (en) 2016-06-29 2022-03-01 Ramot At Tel-Aviv University Ltd. Engineered parasites for delivering protein to the central nervous system (CNS)
CN116509885A (zh) 2016-12-21 2023-08-01 新南创新私人有限公司 增加血管密度的方法
KR20200036912A (ko) * 2017-07-31 2020-04-07 리플렉션 바이오테크놀러지스 리미티드 안과 질환을 위한 세포 모델 및 치료요법

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1964627A (zh) * 2004-06-04 2007-05-16 华盛顿大学 治疗神经病变的方法和组合物
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
US20150259395A1 (en) * 2014-03-17 2015-09-17 Avalanche Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
CN106661591A (zh) * 2014-05-02 2017-05-10 建新公司 用于视网膜及cns基因疗法的aav载体
CN107206105A (zh) * 2014-07-24 2017-09-26 麻省眼耳科医院 色素性视网膜炎的rpgr基因疗法
US20180344719A1 (en) * 2015-10-23 2018-12-06 The Jackson Laboratory Treatment and prevention of ocular neurodegenerative disorder

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
ELIZABETH M. BRYDON ET AL: "AAV-Mediated Gene Augmentation Therapy Restores Critical Functions in Mutant PRPF31+/ iPSC-Derived RPE Cells", 《MOLECULAR THERAPY》, vol. 15, 31 December 2019 (2019-12-31), pages 392 - 402 *
冯雪: "NMNAT1突变致病机理的研究及NMNAT1-LCA9小鼠模型的构建与基因治疗", 《中国博士学位论文全文数据库 医药卫生科技辑》, 15 June 2018 (2018-06-15), pages 47 *
冯雪: "NMNAT1突变致病机理的研究及NMNAT1-LCA9小鼠模型的构建与基因治疗", 《中国博士学位论文全文数据库 医药卫生科技辑》, pages 49 *

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Publication number Publication date
JP2023517929A (ja) 2023-04-27
IL296262A (en) 2022-11-01
WO2021183779A1 (en) 2021-09-16
KR20220152550A (ko) 2022-11-16
MX2022011177A (es) 2022-12-13
CA3174781A1 (en) 2021-09-16
EP4117644A1 (en) 2023-01-18
US20250269068A1 (en) 2025-08-28
US20210299277A1 (en) 2021-09-30
AU2021236233A1 (en) 2022-09-22
BR112022017970A2 (pt) 2022-10-25
US12257320B2 (en) 2025-03-25
EP4117644A4 (en) 2024-08-14

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