Gene therapy offers hope for sickle cell patients by boosting healthy hemoglobin

This study offers expanded access to a gene therapy for people with severe sickle cell disease. The treatment uses a modified virus to deliver genetic material that increases fetal hemoglobin, a healthy type that prevents sickling. Patients receive their own blood stem cells back after a mild chemotherapy, reducing risks like graft-versus-host disease. Early data from 36 patients show no unexpected safety issues and increased fetal hemoglobin.