High-fidelity PAMless base editing of hematopoietic stem cells to treat chronic granulomatous disease
利用高保真无PAM碱基编辑技术对造血干细胞进行治疗慢性肉芽肿病
期刊:Science Translational Medicine
影响因子:14.6
doi:10.1126/scitranslmed.adj6779
Bzhilyanskaya, Vera; Ma, Linyuan; Liu, Siyuan; Fox, Lauren R; Whittaker, Madelynn N; Meis, Ronald J; Choi, Uimook; Lawson, Amanda; Ma, Michelle; Theobald, Narda; Burkett, Sandra; Sweeney, Colin L; Lazzarotto, Cicera R; Tsai, Shengdar Q; Lack, Justin B; Wu, Xiaolin; Dahl, Gary A; Malech, Harry L; Kleinstiver, Benjamin P; De Ravin, Suk See