At present, the only definitive cure for β-thalassemia is a bone marrow transplant (BMT); however, HLA-blood-matched donors are scarcely available. Current therapies undergoing clinical investigation with most potential for therapeutic benefit are the β-globin gene transfer of patient-specific hematopoietic stem cells followed by autologous BMT. Other emerging therapies deliver exogenous regulators of several key modulators of erythropoiesis or iron homeostasis. This review focuses on current approaches for the treatment of hemoglobinopathies caused by disruptions of β-globin. Copyright © 2017 Elsevier Inc. All rights reserved.
|Authors||Guerra A, Musallam KM, Taher AT, Rivella S|
|Journal||Hematology/oncology clinics of North America|
|Publication Date||2018 Apr;32(2):343-352|