Bone marrow transplantation for genetic disorders.

Allogeneic bone marrow transplantation has been successfully utilized to correct the lymphoid and/or hematopoietic abnormalities characterizing a wide array of lethal genetic disorders. Examples include severe combined immunodeficiency, Wiskott-Aldrich syndrome, thalassemia major, sickle cell anemia, and several types of lysosomal storage diseases. Most marrow transplant recipients require preparation with myeloablative doses of chemotherapy, with or without additional radiation therapy, to ensure engraftment of allogeneic marrow. This approach has dramatically changed the long-term outlook for many children with otherwise lethal disorders.