A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells
一种针对大多数DMD患者的CRISPR-Cas9基因敲除策略可恢复hiPSC衍生肌肉细胞中的肌营养不良蛋白功能
期刊:Cell Stem Cell
影响因子:20.4
doi:10.1016/j.stem.2016.01.021
Young, Courtney S; Hicks, Michael R; Ermolova, Natalia V; Nakano, Haruko; Jan, Majib; Younesi, Shahab; Karumbayaram, Saravanan; Kumagai-Cresse, Chino; Wang, Derek; Zack, Jerome A; Kohn, Donald B; Nakano, Atsushi; Nelson, Stanley F; Miceli, M Carrie; Spencer, Melissa J; Pyle, April D