Thalassemia is a fatal hereditary disease in which the body fails to produce normal Haemoglobin. Mutation in genes encoding Alpha and Beta chains are responsible for Thalassemia. It causes slow growth, severe anaemia, organ failure, and jaundice. Patients are dependent on donor blood for a life time. Presently, a bone marrow transplant is considered its treatment, but it is too costly and difficult. However, Haematopoietic Stem Cells have promising potential as they can be used to grow normal bone marrow. Haematopoietic Stem Cells can be collected from a donor. Haematopoiesis will occur either in in-vivo or ex-vivo. Transplantation of fresh Stem Cells or after haematopoiesis will help in the formation of blood precursor cells with normal genotype. In this regard, some amount of immuno-suppressing drugs is also prescribed, which help in overriding problems of mismatch. When newly transplanted cells are accepted by the body, then these cells will give rise to normal blood cells or cells which are genotypically wild. As a result, sustainable and self-sufficient levels of blood cells in the body will be obtained, resulting in the treatment of Thalassemia. Apart from this, Stem Cells can also be used in the treatment of different genetic disorders like Sickle Cell Anaemia, Haemophilia, and many others.
Keywords: Thalassemia, Haematopoietic Stem Cells, Transplantation, Haematopoiesis.