Optimization of prime editing to rewrite the gamma-globin promoters in hematopoietic stem cells from sickle cell patients

Fetal hemoglobin-reactivation is a promising therapy for sickle cell disease. We developed the premiere prime editing strategy that simultaneously introduces multiple gamma-globin-promoting mutations in the gamma-globin promoters of patient hematopoietic stem cells, boosting HbF reactivation and offering alternative therapeutic perspectives.