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Gene Therapy Offers Hope for a Cure

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Scientists are working to develop a gene therapy that may offer a cure for thalassemia. Such a treatment might involve inserting a normal beta globin gene (the gene that is abnormal in this disease) into the patient's stem cells, the immature bone marrow cells that are the precursors of all other cells in the blood.

Another form of gene therapy could involve using drugs or other methods to reactivate the patient's genes that produce fetal hemoglobin - the form of hemoglobin found in fetuses and newborns. Scientists hope that spurring production of fetal hemoglobin will compensate for the patient's deficiency of adult hemoglobin.

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Can Thalassemias Be Prevented?

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As one of five thalassemia centers in the United States designated by the National Institutes of Health, the Children's Hospital Boston thalassemia program provides patients with access to experts in the field and the most cutting-edge clinical trials available in the world. One promising recent study, now awaiting approval by the FDA, tested the effectiveness of a new oral iron chelator, deferisirox. Researchers from Children's Hospital Boston have been at the forefront of thalassemia research and treatment for decades. Important advances in the field that were made here include: • Recognition of the important role of hypertransfusion by Dr. David Nathan, Senior Associate in Hematology, in the 1960s. • Demonstration of effective iron chelation by subcutaneous pump deferoxamine (Desferal ®) by Dr. Richard Proper and David Nathan in the 1970s. • Elucidation of the molecular biology of thalassemia syndromes by Drs. Nathan, YW Kan, Stuart Orkin and others.