Specific DMPK-promoter targeting by CRISPRi reverses myotonic dystrophy type 1-associated defects in patient muscle cells
CRISPRi 靶向特异性 DMPK 启动子可逆转患者肌肉细胞中 1 型强直性肌营养不良症相关缺陷
期刊:Molecular Therapy-Nucleic Acids
影响因子:6.5
doi:10.1016/j.omtn.2023.05.007
Florent Porquet, Lin Weidong, Kévin Jehasse, Hélène Gazon, Maria Kondili, Silvia Blacher, Laurent Massotte, Emmannuel Di Valentin, Denis Furling, Nicolas Albert Gillet, Arnaud François Klein, Vincent Seutin, Luc Willems
