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Bone marrow transplantation for genetic disorders.
Blood Rev. 1990 Jun; 4(2):116-31.BR

Abstract

In 1967, a congenital disorder, severe combined immune deficiency disease, (SCID), was the first condition to be successfully corrected by bone marrow transplantation (BMT) from a histocompatible matched sibling donor. Since then the number of inherited disorders in which BMT has been used has been greatly extended. In preface, it should be stressed that BMT represents only one aspect of the management of genetic disorders which includes first and foremost detection and prevention by antenatal screening. Enzyme replacement treatment and the development of genetic engineering techniques to correct the underlying fault are being actively explored. However, reliable screening programmes are only feasible in a minority of disorders, of which thalassaemia is an example. Enzyme replacement treatment has been largely unsuccessful, and despite considerable advances in the understanding of gene regulation, at present BMT represents the only practice capable of correcting genetic disorders and improving the quality of life of affected individuals.

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Authors+Show Affiliations

Barrett J
Department of Haematology, Royal Postgraduate Medical School, Hammersmith Hospital, London, UK.
McCarthy D
No affiliation info available

MeSH

Bone Marrow TransplantationGenetic Diseases, InbornHematologic DiseasesHumansImmunologic Deficiency SyndromesMetabolism, Inborn ErrorsTissue Donors

Pub Type(s)

Journal Article
Review

Language

eng

PubMed ID

2194596

Citation

Barrett, J, and D McCarthy. "Bone Marrow Transplantation for Genetic Disorders." Blood Reviews, vol. 4, no. 2, 1990, pp. 116-31.
Barrett J, McCarthy D. Bone marrow transplantation for genetic disorders. Blood Rev. 1990;4(2):116-31.
Barrett, J., & McCarthy, D. (1990). Bone marrow transplantation for genetic disorders. Blood Reviews, 4(2), 116-31.
Barrett J, McCarthy D. Bone Marrow Transplantation for Genetic Disorders. Blood Rev. 1990;4(2):116-31. PubMed PMID: 2194596.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Bone marrow transplantation for genetic disorders. AU - Barrett,J, AU - McCarthy,D, PY - 1990/6/1/pubmed PY - 1990/6/1/medline PY - 1990/6/1/entrez SP - 116 EP - 31 JF - Blood reviews JO - Blood Rev VL - 4 IS - 2 N2 - In 1967, a congenital disorder, severe combined immune deficiency disease, (SCID), was the first condition to be successfully corrected by bone marrow transplantation (BMT) from a histocompatible matched sibling donor. Since then the number of inherited disorders in which BMT has been used has been greatly extended. In preface, it should be stressed that BMT represents only one aspect of the management of genetic disorders which includes first and foremost detection and prevention by antenatal screening. Enzyme replacement treatment and the development of genetic engineering techniques to correct the underlying fault are being actively explored. However, reliable screening programmes are only feasible in a minority of disorders, of which thalassaemia is an example. Enzyme replacement treatment has been largely unsuccessful, and despite considerable advances in the understanding of gene regulation, at present BMT represents the only practice capable of correcting genetic disorders and improving the quality of life of affected individuals. SN - 0268-960X UR - https://www.unboundmedicine.com/medline/citation/2194596/Bone_marrow_transplantation_for_genetic_disorders_ L2 - https://linkinghub.elsevier.com/retrieve/pii/0268-960X(90)90035-Q DB - PRIME DP - Unbound Medicine ER -