Gene fix for blood disorder shows early promise in small trial

This early-stage study tests a gene therapy for people with beta-thalassemia major, a severe inherited blood disorder. Doctors take the patient's own stem cells, fix the faulty gene in the lab, and return the corrected cells through an IV. A low dose of busulfan is given beforehand to help the body accept the new cells. The main goals are to check safety and see if the treated cells settle in the bone marrow and start making normal red blood cells.