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| Content Provider | World Health Organization (WHO)-Global Index Medicus |
|---|---|
| Author | Besley, G. T. Wraith, J. E. Hatton, C. E. Arrand, J. E. Fairbairn, L. J. Lebens, G. Lashford, L. S. Mcdermott, R. H. Cooper, A. Dexter, T. M. Arrand, J. R. Hopwood, J. J. Anson, D. S. Spooncer, E. Holt, R. Bellantuono, I. |
| Description | Author Affiliation: Fairbairn LJ ( Department of Experimental Haematology, Paterson Institute for Cancer Research, Christie Hospital National Health Service Trust, Manchester, United Kingdom.); |
| Abstract | Allogeneic bone marrow transplantation is the most effective treatment for Hurler syndrome but, since this therapy is not available to all patients, we have considered an alternative approach based on transfer and expression of the normal gene in autologous bone marrow. A retroviral vector carrying the full-length cDNA for alpha-L-iduronidase has been constructed and used to transduce bone marrow from patients with this disorder. Various gene-transfer protocols have been assessed including the effect of intensive schedules of exposure of bone marrow to viral supernatant and the influence of growth factors. With these protocols, we have demonstrated successful gene transfer into primitive CD34+ cells and subsequent enzyme expression in their maturing progeny. Also, by using long-term bone marrow cultures, we have demonstrated high levels of enzyme expression sustained for several months. The efficiency of gene transfer has been assessed by PCR analysis of hemopoietic colonies as 25-56%. No advantage has been demonstrated for the addition of growth factors or intensive viral exposure schedules. The enzyme is secreted into the medium and functional localization has been demonstrated by reversal of the phenotypic effects of lysosomal storage in macrophages. This work suggests that retroviral gene transfer into human bone marrow may offer the prospect for gene therapy of Hurler syndrome in young patients without a matched sibling donor. |
| ISSN | 00278424 |
| e-ISSN | 10916490 |
| Journal | Proceedings of the National Academy of Sciences of the United States of America |
| Issue Number | 5 |
| Volume Number | 93 |
| Language | English |
| Publisher | National Academy of Sciences |
| Publisher Date | 1996-09-01 |
| Publisher Place | United States |
| Access Restriction | Open |
| Subject Keyword | Genetic Therapy Iduronidase Genetics Mucopolysaccharidosis I Therapy Antigens, CD34 Bone Marrow Enzymology Cells, Cultured DNA Primers Chemistry Gene Expression Genetic Vectors Hematopoietic Stem Cells Molecular Sequence Data Phenotype Time Factors Multidisciplinary |
| Content Type | Text |
| Resource Type | Article |
| Subject | Multidisciplinary |
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