CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease
利用 CRISPR-Cas9 技术编辑 HBG1 和 HBG2 启动子治疗镰状细胞病
期刊:New England Journal of Medicine
影响因子:78.5
doi:10.1056/NEJMoa2215643
Sharma, Akshay; Boelens, Jaap-Jan; Cancio, Maria; Hankins, Jane S; Bhad, Prafulla; Azizy, Marjohn; Lewandowski, Andrew; Zhao, Xiaojun; Chitnis, Shripad; Peddinti, Radhika; Zheng, Yan; Kapoor, Neena; Ciceri, Fabio; Maclachlan, Timothy; Yang, Yi; Liu, Yi; Yuan, Jianping; Naumann, Ulrike; Yu, Vionnie W C; Stevenson, Susan C; De Vita, Serena; LaBelle, James L