Could a One-Time gene fix free thalassemia patients from lifelong transfusions?

This study tests a new gene therapy for adults with transfusion-dependent beta-thalassemia, a severe blood disorder. Doctors take the patient's own blood stem cells, add a healthy gene to make normal hemoglobin, and return the cells. The goal is to reduce or stop the need for regular blood transfusions. The study includes 12 participants and is the first human test of this approach.