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Bone marrow transplantation for genetic and metabolic disorders.
Nouv Rev Fr Hematol. 1989; 31(2):119-23.NR

Abstract

Bone marrow transplantation (BMT) was primarily used for the treatment of hematologic malignancies and aplastic anemias. BMT has been successfully applied as curative treatment in a series of lethal congenital disorders but among more than 1000 inherited metabolic diseases only 20 of them can be corrected by transplantation. The inborn error must be expressed in the stem cells. The genetic disorders that can be cured include congenital immune deficiencies, osteopetrosis, and congenital lysosomal storage diseases, mucopolysaccharidosis and lipidosis. BMT with an HLA matched donor is successful in 60% of patients with severe combined immune deficiency and Wiskott Aldrich syndrome. Results are good in osteopetrosis if BMT is performed early. Encouraging results have been reported in Hurler disease and in Gaucher disease.

Authors+Show Affiliations

Unité d'Hématologie Pédiatrique, Hôpital Saint-Louis, Paris, France.No affiliation info availableNo affiliation info available

Pub Type(s)

Journal Article
Review

Language

eng

PubMed ID

2505227

Citation

Schaison, G, et al. "Bone Marrow Transplantation for Genetic and Metabolic Disorders." Nouvelle Revue Francaise D'hematologie, vol. 31, no. 2, 1989, pp. 119-23.
Schaison G, Bordigoni P, Leverger G. Bone marrow transplantation for genetic and metabolic disorders. Nouv Rev Fr Hematol. 1989;31(2):119-23.
Schaison, G., Bordigoni, P., & Leverger, G. (1989). Bone marrow transplantation for genetic and metabolic disorders. Nouvelle Revue Francaise D'hematologie, 31(2), 119-23.
Schaison G, Bordigoni P, Leverger G. Bone Marrow Transplantation for Genetic and Metabolic Disorders. Nouv Rev Fr Hematol. 1989;31(2):119-23. PubMed PMID: 2505227.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Bone marrow transplantation for genetic and metabolic disorders. AU - Schaison,G, AU - Bordigoni,P, AU - Leverger,G, PY - 1989/1/1/pubmed PY - 1989/1/1/medline PY - 1989/1/1/entrez SP - 119 EP - 23 JF - Nouvelle revue francaise d'hematologie JO - Nouv Rev Fr Hematol VL - 31 IS - 2 N2 - Bone marrow transplantation (BMT) was primarily used for the treatment of hematologic malignancies and aplastic anemias. BMT has been successfully applied as curative treatment in a series of lethal congenital disorders but among more than 1000 inherited metabolic diseases only 20 of them can be corrected by transplantation. The inborn error must be expressed in the stem cells. The genetic disorders that can be cured include congenital immune deficiencies, osteopetrosis, and congenital lysosomal storage diseases, mucopolysaccharidosis and lipidosis. BMT with an HLA matched donor is successful in 60% of patients with severe combined immune deficiency and Wiskott Aldrich syndrome. Results are good in osteopetrosis if BMT is performed early. Encouraging results have been reported in Hurler disease and in Gaucher disease. UR - https://www.unboundmedicine.com/medline/citation/2505227/Bone_marrow_transplantation_for_genetic_and_metabolic_disorders_ L2 - https://www.diseaseinfosearch.org/result/7171 DB - PRIME DP - Unbound Medicine ER -