In vivo genome editing of human hematopoietic stem cells for treatment of blood disorders by mRNA delivery
Saijuan Xu, Dan Liang, Qiudao Wang, Yan Cheng, Da Xie, Gui Yang, Haokun Zhang, Changrui Feng, Feiyan Zhao, Wendan Ren, Gongrui Sun, Yang Yang, Lin Li, Yongrong Lai, Bin Fu, , Yuxuan Wu
This paper develops an antibody-free lipid nanoparticle system for in vivo mRNA delivery to human HSCs, enabling efficient base editing of HBG to treat blood disorders like beta-thalassemia and sickle cell disease.
Nature Biomedical Engineering · Aug 12, 2025Peer-reviewedBioengineering