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

Molecules of sickle-cell hemoglobin stick to one another ... often resulting in a frameshift One form of beta-thalassemia Deletion One or more nucleotides is "skipped" during replication or ...

“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 ...

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