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Issue 1054 coverCooley's Anemia: Eighth Symposium Volume 1054 published November 2005
Ann. N.Y. Acad. Sci. 1054: 1–10 (2005). doi: 10.1196/annals.1345.001
Copyright © 2005 by the New York Academy of Sciences
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Articles by NATHAN, D. G.
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Articles by NATHAN, D. G.
Thalassemia: The Continued Challenge

DAVID G. NATHAN

Dana-Farber Cancer Institute, Boston, MA 02115, USA, and Harvard Medical School, Boston, MA 02115, USA

Address for correspondence: David G. Nathan, M.D., President Emeritus, Dana-Farber Cancer Institute, 44 Binney Street, Boston, MA 02115. Voice: 617-632-2155; fax: 617-632-2161. David_Nathan{at}dfci.harvard.edu

This overview describes the history of transfusion therapy and consequent iron overload in thalassemia. It emphasizes the importance of measurement of hepatic iron and reviews the history of chelation therapy. It briefly describes the discoveries of the genetic basis of thalassemia and the application of that knowledge in prenatal diagnosis. The review goes on to emphasize pharmaceutical efforts to induce fetal hemoglobin synthesis in thalassemic red cells and ends with a discussion of oral iron chelators, stem cell transplant, and the status of gene therapy.

Key Words: thalassemia • chelation therapy • transfusion therapy • hepatic iron • genetics • prenatal diagnosis • stem cell transplantation • gene therapy




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{beta}-Thalassemia
N. Engl. J. Med., September 15, 2005; 353(11): 1135 - 1146.
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