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    Long-term in vitro correction of alpha-L-iduronidase deficiency (Hurler syndrome) in human bone marrow.

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    Authors
    Fairbairn, Leslie J
    Lashford, Linda S
    Spooncer, Elaine
    McDermott, R H
    Lebens, G
    Arrand, Jane E
    Arrand, John R
    Bellantuono, Ilaria
    Holt, R
    Hatton, C E
    Cooper, A
    Besley, G T
    Wraith, J Ed
    Anson, D S
    Hopwood, J J
    Dexter, T Michael
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    Affiliation
    Department of Experimental Haematology, Paterson Institute for Cancer Research, Christie Hospital National Health Service Trust, Manchester, United Kingdom.
    Issue Date
    1996-03-05
    
    Metadata
    Show full item record
    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.
    Citation
    Long-term in vitro correction of alpha-L-iduronidase deficiency (Hurler syndrome) in human bone marrow. 1996, 93 (5):2025-30 Proc. Natl. Acad. Sci. U.S.A.
    Journal
    Proceedings of the National Academy of Sciences of the United States of America
    URI
    http://hdl.handle.net/10541/95890
    PubMed ID
    8700879
    Type
    Article
    Language
    en
    ISSN
    0027-8424
    Collections
    All Paterson Institute for Cancer Research

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