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

“Instead, we use a viral vector to deliver a new gene that will make a healthy hemoglobin—a beta hemoglobin—into the stem cell. This is like coding new instructions into the cell. The old instructions ...

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 ...

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