“In this therapy, we do not change or edit the genes that cause sickle cell disease,” Kanter says. “Instead, we use a viral vector to deliver a new gene that will make a healthy hemoglobin—a beta ...

An essential factor in distinguishing thalassemia phenotypes is transfusion dependence. While beta-thalassemia major requires ...

Orkin focused his attention on the hemoglobin gene, which is mutated in people with sickle cell anemia and another set of ...