2区 · 医学
Article
作者: Woodard, Kaitly J. ; Hsieh, Matthew M. ; Shea, Devlin ; Neel, Michael D. ; Ryu, Byoung Y. ; Lazzarotto, Cicera R. ; Uchida, Naoya ; Métais, Jean-Yves ; Bauer, Daniel E. ; Zeng, Jing ; Porter, Shaina N. ; Tisdale, John F. ; Fowler, Stephanie C. ; Doerfler, Phillip A. ; Mayuranathan, Thiyagaraj ; Tsai, Shengdar Q. ; Weiss, Mitchell J. ; Katta, Varun ; Yao, Yu ; Wolfe, Scot A. ; Perry, S. Scott ; Luk, Kevin ; Keriwala, Sagar ; Peters, Samuel T. ; Sharma, Akshay ; Pruett-Miller, Shondra ; Wu, Yuxuan
Key PointsCas9 editing of the γ-globin gene promoters in hematopoietic stem cells (HSCs) increases red cell HbF by ≤40%. No deleterious effects on hematopoiesis or off-target mutations were detected 16 weeks after xenotransplantation of edited HSCs.