Hematopoietic Stem-Cell Transplantation for the Treatment of Beta Thalassemia

Beta thalassemia is caused by a large number of various genetic mutations or deletions of theglobin gene which cause a reduction in the synthesis and accumulation of the beta-globinpolypeptide (1-3). This gives rise to an imbalance of the beta and alpha globin chains, which inturn, leads to the formation of significant amounts of unstable alpha globin tetramers (4-6).These unstable tetramers give rise to oxidative damage of the red cell membrane and a decreasein its lifespan. Thalassemias affect the populations of Mediterranean and South Asian Ancestry.Homozygous beta thalassemia represents the most frequent form of thalassemia in the US withan estimatd total number of 1,000 patients (7,8).