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公开(公告)号:US20230159609A1
公开(公告)日:2023-05-25
申请号:US17995880
申请日:2020-07-10
申请人: Sorbonne Université , Centre National de la Recherche Scientifique , INSERM (Institut National de la Santé et de la Recherche Médicale) , Ruhr-Universität Bochum , Centrre Hospitalier National d'Ophtalmologie Quinze-Vingts
发明人: Deniz DALKARA , Cardillia-Joe SIMON , Stefan HERLITZE , José-Alain SAHEL , Isabelle AUDO , Serge PICAUD , Stéphane Bertin
IPC分类号: C07K14/705 , C12N15/86 , C07K14/005 , A61P27/02 , A61P25/00
CPC分类号: C07K14/705 , C12N15/86 , C07K14/005 , A61P27/02 , A61P25/00 , C12N2750/14143 , C12N2750/14145 , C12N2750/14171 , C12N2750/14122 , A61K48/00
摘要: The present invention concerns a new gene therapy approach to increase light-sensitivity in degenerating cones in advanced stages of rod-cone dystrophy (RCD) mediated by G-protein-gated-K+ channel (GIRK), in particular GIRK2, activated by G proteins recruited by cone opsin expressed in degenerating cones.