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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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