Dissecting ELANE neutropenia pathogenicity by human HSC gene editingCELL STEM CELLIF20.42021-05-0128OA
BCL11A enhancer edited hematopoietic stem cells persist in rhesus monkeys without toxicityBCL11A增强子编辑的造血干细胞在恒河猴中持续存在而没有毒性JOURNAL OF CLINICAL INVESTIGATIONIF13.62020-11-0953OA
Highly efficient therapeutic gene editing of human hematopoietic stem cells人类造血干细胞的高效治疗性基因编辑NATURE MEDICINEIF502019-03-25365OAAI