What research in Thalassemia is taking place?
Treatment today is more advanced then what it was. Scientists are working on ways to eliminate excess iron from the body in order to check or delay iron overload. They are developing and testing the effectiveness of oral iron-chelating drugs (L1), which could significantly simplify treatment. They are also developing an helpful type of gene therapy that may one day present a cure for Thalassemia. The results from the L1 have been excellent except of one particular side effect called “Neutropenia” which reduces the ability of the body to cope with infections so the patient has an increased threat at infections that can be fatal. Gene therapy may 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 may involve using drugs or other methods to reactivate the patient’s genes for fetal hemoglobin. All humans prod
