Gene therapy treats sickle cell disease by fixing the problem at the genetic level, but only in the blood-forming cells. Instead of using donor cells, gene therapy uses the patient’s own stem cells.

For decades, sickle cell disease has been managed, not cured. Pain crises, frequent hospital visits, blood transfusions, organ damage, and a shortened quality of life have been accepted as “normal” for people living with the condition. Today, stem cell transplant, also called bone marrow transplant, is the only proven cure for sickle cell disease.