Gene Therapy for Hemoglobinopathies

Beta hemoglobinopathies such as sickle cell disease (SCD) and β-thalassemia (BT) are the most common monogenic diseases worldwide. Both diseases are associated with significant morbidity and mortality. Because patients require lifelong follow-up and care, it also poses a serious burden in health services. Blood transfusions and/or drug therapy ameliorate the signs and symptoms of the disorders but are not curative. Allogeneic hematopoietic cell transplantation (HCT) is currently the only cure but it has several limitations including the paucity of human leukocyte antigen-matched related donors and a high risk of adverse events.
Source: Transfusion and Apheresis Science - Category: Hematology Authors: Source Type: research