• Hematol. Oncol. Clin. North Am. · Dec 2010

    Review

    Hemoglobin gene therapy for β-thalassemia.

    • Arthur Bank.
    • Columbia University, New York, NY, USA. ab13@columbia.edu
    • Hematol. Oncol. Clin. North Am. 2010 Dec 1; 24 (6): 1187-201.

    AbstractAllogeneic stem cell transplantation currently is the only curative option for severe β-thalassemia and sickle cell disease. Human globin gene therapy with autotransplantation of transduced human hematopoietic stem cells is an exciting alternative approach to a potential cure. One patient with thalassemia has recently been reported to have clinical benefit after lentiviral human β-globin gene therapy. He has not required blood transfusions for almost 2 years. Most of the patient's gene correction and new human β-globin gene expression is caused by the expansion of a single clone in which the corrective transgene is inserted into an Hmga2 gene.Copyright © 2010 Elsevier Inc. All rights reserved.

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