- 专利标题： VECTORS ENCODING A GLUCOSE-6-PHOSPHATASE (G6PASE-A) FOR GENE THERAPY
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申请号： US17926709申请日： 2021-05-21
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公开(公告)号： US20230203534A1公开(公告)日： 2023-06-29
- 发明人: Louisa JAUZE , Fabienne RAJAS , Giuseppe RONZITTI
- 申请人： GENETHON , INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM) , UNIVERSITE D'EVRY VAL D'ESSONNE , UNIVERSITE CLAUDE BERNARD LYON 1
- 申请人地址： FR EVRY-COURCOURONNES
- 专利权人： GENETHON,INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM),UNIVERSITE D'EVRY VAL D'ESSONNE,UNIVERSITE CLAUDE BERNARD LYON 1
- 当前专利权人： GENETHON,INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM),UNIVERSITE D'EVRY VAL D'ESSONNE,UNIVERSITE CLAUDE BERNARD LYON 1
- 当前专利权人地址： FR EVRY-COURCOURONNES
- 优先权： EP 305540.5 2020.05.22
- 国际申请： PCT/EP2021/063728 2021.05.21
- 进入国家日期： 2022-11-21
- 主分类号： C12N15/86
- IPC分类号： C12N15/86 ; C07K14/81 ; C12N9/16
摘要：
The invention relates to an adeno-associated virus (AAV) vector comprising a nucleic acid construct for the expression of a glucose-6-phosphatase-a (G6Pase-a) in a cell, the construct comprising a nucleic acid sequence encoding the G6Pase-a, wherein the nucleic acid sequence encoding the G6Pase-a is operably linked to a human alpha-1 antitrypsin (hAAT) promoter, a cell transformed with the vector of the invention, a composition comprising the vector or the cell of the invention, and the use thereof.
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