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    Retrovirally mediated correction of bone marrow-derived mesenchymal stem cells from patients with mucopolysaccharidosis type I.

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    Authors
    Baxter, Melissa A
    Wynn, Robert F
    Deakin, Jon A
    Bellantuono, Ilaria
    Edington, Kirsten G
    Cooper, Alan
    Besley, Guy T N
    Church, Heather J
    Wraith, J Ed
    Carr, Trevor F
    Fairbairn, Leslie J
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    Affiliation
    Department of Haematology, Royal Manchester Children's Hospital, United Kingdom.
    Issue Date
    2002-03-01
    
    Metadata
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    Abstract
    We have investigated the utility of bone marrow-derived mesenchymal stem cells (MSCs) as targets for gene therapy of the autosomal recessive disorder mucopolysaccharidosis type IH (MPS-IH, Hurler syndrome). Cultures of MSCs were initially exposed to a green fluorescent protein-expressing retrovirus. Green fluorescent protein-positive cells maintained their proliferative and differentiation capacity. Next we used a vector encoding alpha-L-iduronidase (IDUA), the enzyme that is defective in MPS-IH. Following transduction, MPS-IH MSCs expressed high levels of IDUA and secreted supernormal levels of this enzyme into the extracellular medium. Exogenous IDUA expression led to a normalization of glycosaminoglycan storage in MPS-IH cells, as evidenced by a dramatic decrease in the amount of (35)SO(4) sequestered within the heparan sulfate and dermatan sulfate compartments of these cells. Finally, gene-modified MSCs were able to cross-correct the enzyme defect in untransduced MPS-IH fibroblasts via protein transfer.
    Citation
    Retrovirally mediated correction of bone marrow-derived mesenchymal stem cells from patients with mucopolysaccharidosis type I. 2002, 99 (5):1857-9 Blood
    Journal
    Blood
    URI
    http://hdl.handle.net/10541/84055
    DOI
    http://dx.doi.org/10.1182/blood.V99.5.1857
    PubMed ID
    11861306
    Type
    Article
    Language
    en
    ISSN
    0006-4971
    ae974a485f413a2113503eed53cd6c53
    http://dx.doi.org/10.1182/blood.V99.5.1857
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