Bone marrow transplantation for genetic diseases.
Bone marrow transplantation is the treatment of choice for a number of genetic diseases. Recently, bone marrow transplantation has been increasingly used for erythroid disorders, such as thalassemia and sickle cell anemia. A number of inherited metabolic disorders (i.e., storage diseases, leukodystrophies, and the like) may be corrected with a marrow transplant. Successful correction of genetic diseases with allogeneic bone marrow transplantation lays the groundwork for the use of specific gene therapy.
Lenarsky
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University of Southern California School of Medicine.
Kohn
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Weinberg
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Parkman
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D016026:Bone Marrow Transplantation; D001927:Brain Diseases / Q000601:surgery; D030342:Genetic Diseases, Inborn / Q000601:surgery*; D006402:Hematologic Diseases / Q000235:genetics / Q000601:surgery; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000235:genetics / Q000601:surgery; D009083:Mucopolysaccharidoses / Q000601:surgery; D010022:Osteopetrosis / Q000628:therapy; D013577:Syndrome