Gene editing could cure sickle cell and thalassemia in new trial

This early-stage trial tests a gene-editing treatment for people with severe sickle cell disease or beta-thalassemia. The approach uses the patient's own blood stem cells, which are edited to boost fetal hemoglobin—a healthy type that can replace the defective hemoglobin. Up to 10 participants aged 13-40 will receive the edited cells after mild chemotherapy, with the goal of curing their condition without the need for a donor or lifelong medication.