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

作　　者：Weiqi Hong Mengyuan Huang Yuquan Wei Xiawei Wei 

机构地区：[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

出　　处：《Science Bulletin》2019年第21期1562-1564,共3页科学通报（英文版）

摘　　要：Site-specific gene editing is of great importance in precise medicine. Two conventional genome editing methods, Zine finger nucleases (ZFNs) and transcription activator-like effector nucleases(TALENs), are based on protein-DNA recognition, with tedious work in constructing target protein [1,2]. Developed from immune response of bacteria, CRISPR/Cas9 has been widely investigated as a promising tool for therapeutic genome editing in clinical settings nowadays[3,4].

关 键 词：CRISPR/Cas9 TALENs ZFNs 

分 类 号：Q78[生物学—分子生物学]