- 专利标题: Selective gene therapy expression system
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申请号: US14782396申请日: 2014-04-10
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公开(公告)号: US09981049B2公开(公告)日: 2018-05-29
- 发明人: Ana Maria Buj Bello , Isabelle Richard
- 申请人: GENETHON , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE
- 申请人地址: FR Evry FR Paris
- 专利权人: GENETHON,CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE
- 当前专利权人: GENETHON,CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE
- 当前专利权人地址: FR Evry FR Paris
- 代理机构: Seed IP Law Group LLP
- 优先权: FR1353306 20130411
- 国际申请: PCT/FR2014/050866 WO 20140410
- 国际公布: WO2014/167253 WO 20141016
- 主分类号: A61K48/00
- IPC分类号: A61K48/00 ; C12N9/16 ; C12N9/64 ; A61K38/46 ; A61K38/48 ; C12N15/86 ; C07K14/47
摘要:
The present invention relates to an expression system for systemic administration comprising a sequence encoding a protein, said expression system allowing: the expression at a therapeutically acceptable level of the protein in the target tissues including skeletal muscles and/or the peripheral nervous tissue; and the expression at toxically acceptable level of the protein in tissues other than the target tissues, especially in the heart.
公开/授权文献
- US20160058890A1 SELECTIVE GENE THERAPY EXPRESSION SYSTEM 公开/授权日:2016-03-03
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