Reduction of BCL11A in hematopoietic stem cells through gene editing: new strategy to ameliorate the severe β-globin disorders sickle cell disease

Affiliations

• ¹ Laboratory of Aging Research and Cancer Drug Target, State Key Laboratory of Biotherapy, National Clinical Research Center for Geriatrics, West China Hospital, Sichuan University, Chengdu 610041, China.
• ² Laboratory of Aging Research and Cancer Drug Target, State Key Laboratory of Biotherapy, National Clinical Research Center for Geriatrics, West China Hospital, Sichuan University, Chengdu 610041, China. Electronic address: xiaweiwei@scu.edu.cn.