title: Bone marrow transplantation for genetic diseases.
abstract: 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.
authors.affiliation: University of Southern California School of Medicine.
authors.forename: C
authors.initials: C
authors.lastname: Lenarsky
authors.forename: D B
authors.initials: DB
authors.lastname: Kohn
authors.forename: K I
authors.initials: KI
authors.lastname: Weinberg
authors.forename: R
authors.initials: R
authors.lastname: Parkman
mesh_terms: 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