Thelansis

  β-thalassemia are monogenic disorders usually caused by reduced or absent synthesis of the βglobin chain, one of the main components of adult hemoglobin (HbA, α2β2. This results in a relative increase in the unattached αglobin chains (α/β-chain imbalance) that form insoluble Hemi-chromes in the erythrocyte progenitors. The Hemi-chromes damage the erythrocyte membrane, leading to severe intramedullary erythrocyte apoptosis (ineffective erythropoiesis, IE) and severely shortened red blood cell (RBC) life span due to extra-medullary hemolysis, leading to severe anemia. ·        Transfusion-dependent β-thalassemia (TDT) is the most severe form of the disease, characterized by severe anemia, and requires lifelong, regular blood transfusions to maintain Hb levels. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload ·        In the USA the estimated β-thalassemia cases range ...