Base Editing of the HBG Promoter Induces Potent Fetal Hemoglobin Expression with No Detectable Off-target Mutations in Human HSCs
Wenyan Han, Hou-Yuan Qiu, Shangwu Sun, Zhi-Can Fu, Guo-Quan Wang, Xiaowen Qian, Lijie Wang, Xiaowen Zhai, Jia Wei, Yichuan Wang, Yi-Lin Guo, Guo-Hua Cao, Rui-Jin Ji, Yi-Zhou Zhang, Hongxia Ma, Hongsheng Wang, Mingli Zhao, Jing Wu
PDF4.9 MB
- EN