Targeted genome editing in vivo corrects a Dmd duplication restoring wild-type dystrophin expression
体内靶向基因组编辑可纠正Dmd基因重复,从而恢复野生型肌营养不良蛋白的表达。
期刊:EMBO Molecular Medicine
影响因子:9
doi:10.15252/emmm.202013228
Eleonora Maino ,Daria Wojtal ,Sonia L Evagelou ,Aiman Farheen ,Tatianna W Y Wong ,Kyle Lindsay ,Ori Scott ,Samar Z Rizvi ,Elzbieta Hyatt ,Matthew Rok ,Shagana Visuvanathan ,Amanda Chiodo ,Michelle Schneeweiss ,Evgueni A Ivakine ,Ronald D Cohn