Читать книгу The SAGE Encyclopedia of Stem Cell Research - Группа авторов - Страница 160

Allogeneic bone marrow transplants have also been used in the treatment of hereditary blood conditions, including beta thalassemia, aplastic anemia, Fanconi anemia, and sickle cell anemia; however, due to the risks associated with this, this is rarely used unless the condition is fatal.

Thalassemia is a disorder commonly found in Mediterranean people and is of two types: alpha and beta. In beta thalassemia, the beta globulin chain is absent in the hemoglobin and it leads to severe lifelong anemia, requiring frequent transfusions and thus causes a poorer quality of life.

Sickle cell anemia is another hereditary condition where the glutamic acid is substituted by valine in the beta globulin chain of hemoglobin, and this leads to abnormal-shaped RBCs and the vaso-occlusive crises associated with the condition. The only known cure for sickle cell disease in children is HSC transplant, but due to the difficulty in finding an HLA match for donors, this is a rare treatment.

There have been successful BMTs in such patients, however the pool of possible HLA-matched donors is small, and there have been attempts to use induced pluripotent stem cells for the treatment of thalassemia and sickle cell anemia.