Bone marrow transplantation for genetic diseases

Hematol Oncol Clin North Am. 1990 Jun;4(3):589-602.

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.

Publication types

  • Review

MeSH terms

  • Bone Marrow Transplantation*
  • Brain Diseases / surgery
  • Genetic Diseases, Inborn / surgery*
  • Hematologic Diseases / genetics
  • Hematologic Diseases / surgery
  • Humans
  • Immunologic Deficiency Syndromes / genetics
  • Immunologic Deficiency Syndromes / surgery
  • Mucopolysaccharidoses / surgery
  • Osteopetrosis / therapy
  • Syndrome