Gene fix for thalassemia: first human test shows promise

This early-stage study tests a gene therapy for adults with β-thalassemia major, a severe blood disorder requiring frequent transfusions. Doctors take the patient's own stem cells, fix the faulty gene, and return the corrected cells via an IV. A low dose of busulfan prepares the body to accept the new cells. The main goals are to check safety and see if the treated cells can produce normal red blood cells.