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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 This article has been cited by other articles:
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