Therapeutically relevant engraftment of a CRISPR-Cas9-edited HSC-enriched population with HbF reactivation in nonhuman primates
在非人类灵长类动物中，通过 CRISPR-Cas9 编辑的富含 HSC 的细胞群进行治疗相关的植入，并重新激活 HbF
期刊:Science Translational Medicine
影响因子:15.8
doi:10.1126/scitranslmed.aaw3768
Olivier Humbert, Stefan Radtke, Clare Samuelson, Ray R Carrillo, Anai M Perez, Sowmya S Reddy, Christopher Lux, Sowmya Pattabhi, Lauren E Schefter, Olivier Negre, Ciaran M Lee, Gang Bao, Jennifer E Adair, Christopher W Peterson, David J Rawlings, Andrew M Scharenberg, Hans-Peter Kiem
