Epitope editing enables chemotherapy-free stem cell transplant for sickle cell disease

Scientists used epitope editing of the KIT protein to enable antibody-based, non-genotoxic conditioning for stem cell transplantation, avoiding the need for chemotherapy. The technique selectively enriches therapeutically edited BCL11A haematopoietic stem cells and boosts fetal haemoglobin production. The approach could transform treatment of sickle cell disease and -thalassaemia.