Emerging Therapies
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W.B. Saunders
Abstract
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. © 2017 Elsevier Inc.
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Keywords
Gene transfer, Hemichromes, New therapies, Trap ligands, Β-globin, Β-thalassemia, Animals, Beta-globins, Beta-thalassemia, Clinical trials as topic, Combined modality therapy, Disease management, Drug discovery, Drug evaluation, preclinical, Erythropoiesis, Humans, Beta globulin, Hemoglobin beta chain, Autologous bone marrow transplantation, Beta thalassemia, Drug development, Experimental therapy, Gene therapy, Hematopoietic stem cell transplantation, Human, Nonhuman, Preclinical study, Priority journal, Randomized controlled trial (topic), Review, Animal, Blood, Clinical trial (topic), Genetics, Multimodality cancer therapy, Procedures