发明公开
- 专利标题: PROMOTERS AND USES THEREOF
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申请号: EP16806078.8申请日: 2016-12-05
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公开(公告)号: EP3384036A1公开(公告)日: 2018-10-10
- 发明人: DALKARA, Deniz , PICAUD, Serge , DESROSIERS, Melissa , SAHEL, José-Alain , DUEBEL, Jens , BEMELMANS, Alexis , ROSKA, Botond
- 申请人: Sorbonne Université , INSERM (Institut National de la Santé et de la Recherche Médicale) , Centre National de la Recherche Scientifique , Friedrich Miescher Institute for Biomedical Research
- 申请人地址: 21 rue de l'École de Médecine 75006 Paris FR
- 专利权人: Sorbonne Université,INSERM (Institut National de la Santé et de la Recherche Médicale),Centre National de la Recherche Scientifique,Friedrich Miescher Institute for Biomedical Research
- 当前专利权人: Sorbonne Université,INSERM (Institut National de la Santé et de la Recherche Médicale),Centre National de la Recherche Scientifique,Friedrich Miescher Institute for Biomedical Research
- 当前专利权人地址: 21 rue de l'École de Médecine 75006 Paris FR
- 代理机构: Cabinet Becker et Associés
- 优先权: EP15306932 20151204
- 国际公布: WO2017093566 20170608
- 主分类号: C12N15/861
- IPC分类号: C12N15/861
摘要:
The present invention relates to a nucleic acid sequence derived from the regulatory region of the human gamma-synuclein gene and having a promoter activity in retinal ganglion cells. The present invention also relates to expression cassettes or vectors comprising said promoter operably linked to a nucleic acid sequence encoding a polypeptide of interest as well as viral particles or host cells comprising said expression cassette or vector. The present invention also relates to the use of said expression cassettes, vectors, viral particles or cells in the treatment of ocular disease, in particular ocular disease associated with retinal ganglion cell or photoreceptor cell degeneration.
公开/授权文献
- EP3384036B1 PROMOTERS AND USES THEREOF 公开/授权日:2021-02-24
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