Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease
开发和IND申报研究：一种新型Cas9基因编辑自体CD34+细胞疗法，用于诱导胎儿血红蛋白以治疗镰状细胞病。
期刊:Molecular Therapy
影响因子:12.1
doi:10.1016/j.ymthe.2024.07.022
Varun Katta,Kiera O'Keefe,Yichao Li,Thiyagaraj Mayuranathan,Cicera R Lazzarotto,Rachael K Wood,Rachel M Levine,Alicia Powers,Kalin Mayberry,Garret Manquen,Yu Yao,Jingjing Zhang,Yoonjeong Jang,Nikitha Nimmagadda,Erin A Dempsey,GaHyun Lee,Naoya Uchida,Yong Cheng,Frank Fazio,Tim Lockey,Mike Meagher,Akshay Sharma,John F Tisdale,Sheng Zhou,Jonathan S Yen,Mitchell J Weiss,Shengdar Q Tsai
