title: Bone marrow transplantation for inherited diseases.
abstract: Allogeneic bone marrow transplantation has been used successfully for the treatment of a variety of inherited diseases. The goal of transplantation in this setting is to provide a sufficient degree of sustained marrow engraftment to allow longterm amelioration of the inherited disease phenotype. Many factors influence the likelihood of achieving this goal, including donor availability, conditioning regimen, marrow processing, and the nature and extent of progression of the disease. For many inherited diseases early diagnosis is imperative because the outcome of transplantation is more favorable when performed prior to the development of significant organ damage from the disease, its complications, or treatment. Although the results of bone marrow transplantation are good for some inherited diseases and are improving for others, significant problems remain such as donor availability, conditioning regimen toxicity, graft failure, and graft-versus-host disease. This review describes some of the unique features of bone marrow transplantation for inherited diseases and discusses recent advances in this area.
authors.affiliation: Division of Pediatric Bone Marrow Transplantation, University of California at San Francisco 94143, USA.
authors.forename: A S
authors.initials: AS
authors.lastname: O'Marcaigh
authors.forename: M J
authors.initials: MJ
authors.lastname: Cowan
mesh_terms: D000755:Anemia, Sickle Cell / Q000628:therapy; D016026:Bone Marrow Transplantation; D005199:Fanconi Anemia / Q000628:therapy; D030342:Genetic Diseases, Inborn / Q000628:therapy*; D006402:Hematologic Diseases / Q000628:therapy*; D006801:Humans; D016464:Lysosomal Storage Diseases / Q000628:therapy; D016511:Severe Combined Immunodeficiency / Q000628:therapy; D014184:Transplantation, Homologous; D017086:beta-Thalassemia / Q000628:therapy