Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease
人类造血干细胞中 β 珠蛋白基因校正的开发可作为镰状细胞病的潜在持久治疗方法
期刊:Science Translational Medicine
影响因子:15.8
doi:10.1126/scitranslmed.abf2444
Annalisa Lattanzi, Joab Camarena, Premanjali Lahiri, Helen Segal, Waracharee Srifa, Christopher A Vakulskas, Richard L Frock, Josefin Kenrick, Ciaran Lee, Narae Talbott, Jason Skowronski, M Kyle Cromer, Carsten T Charlesworth, Rasmus O Bak, Sruthi Mantri, Gang Bao, David DiGiusto, John Tisdale, J Fr
