SCIENCE · Jul 11, 2026 Many CRISPR-based sickle cell disease clinical trials primarily edit the BCL11A gene or its enhancer to reactivate fetal hemoglobin rather than directly repairing the HBB mutation. NOT BS
HEALTH · Jul 11, 2026 Ex vivo CRISPR-based therapies for sickle cell disease typically require myeloablative conditioning with busulfan before reinfusion of edited hematopoietic stem cells. NOT BS