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Science 13 June 1986:
Vol. 232. no. 4756, pp. 1373 - 1378
DOI: 10.1126/science.3520819
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Articles

Science, Vol 232, Issue 4756, 1373-1378
Copyright © 1986 by American Association for the Advancement of Science

articles

The application of bone marrow transplantation to the treatment of genetic diseases

R Parkman

Genetic diseases can be treated by transplantation of either normal allogeneic bone marrow or, potentially, autologous bone marrow into which the normal gene has been inserted in vitro (gene therapy). Histocompatible allogeneic bone marrow transplantation is used for the treatment of genetic diseases whose clinical expression is restricted to lymphoid or hematopoietic cells. The therapeutic role of bone marrow transplantation in the treatment of generalized genetic diseases, especially those affecting the central nervous system, is under investigation. The response of a generalized genetic disease to allogeneic bone marrow transplantation may be predicted by experiments in vitro. Gene therapy can be used only when the gene responsible for the disease has been characterized. Success of gene therapy for a specific genetic disease may be predicted by its clinical response to allogeneic bone marrow transplantation.


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Bone Marrow Transplantation for Sickle Cell Disease.
M. C. Walters, M. Patience, W. Leisenring, J. R. Eckman, J. P. Scott, W. C. Mentzer, S. C. Davies, K. Ohene-Frempong, F. Bernaudin, D. C. Matthews, et al. (1996)
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Gene Therapy in Peripheral Blood Lymphocytes and Bone Marrow for ADA- Immunodeficient Patients.
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Science 270, 470-475
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Chimerism after Liver Transplantation for Type IV Glycogen Storage Disease and Type 1 Gaucher's Disease.
T. E. Starzl, A. J. Demetris, M. Trucco, C. Ricordi, S. Ildstad, P. I. Terasaki, N. Murase, R. S. Kendall, M. Kocova, W. A. Rudert, et al. (1993)
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An in vivo model of somatic cell gene therapy for human severe combined immunodeficiency.
G Ferrari, S Rossini, R Giavazzi, D Maggioni, N Nobili, M Soldati, G Ungers, F Mavilio, E Gilboa, and C Bordignon (1991)
Science 251, 1363-1366
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Bone Marrow Transplantation and Intensive Care Units: A Need for Integration.
R. Parkman (1987)
J Intensive Care Med 2, 297-298
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Science. ISSN 0036-8075 (print), 1095-9203 (online)