In vivo genome editing of human hematopoietic stem cells for treatment of blood disorders by mRNA delivery
Saijuan Xu, Dan Liang, Qiudao Wang…·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.