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Blood, 1 March 2002, Vol. 99, No. 5, pp. 1857-1859
BRIEF REPORT
Retrovirally mediated correction of bone marrow-derived
mesenchymal stem cells from patients with mucopolysaccharidosis
type I
Melissa A. Baxter,
Robert
F. Wynn,
Jonathan A. Deakin,
Ilaria Bellantuono,
Kirsten G. Edington,
Alan Cooper,
Guy T. N. Besley,
Heather J. Church,
J. Ed Wraith,
Trevor F. Carr, and
Leslie J. Fairbairn
From the Departments of Haematology and Metabolic
Medicine, Royal Manchester Children's Hospital; and CRC Gene Therapy
Group and Medical Oncology, Paterson Institute for Cancer Research,
Christie Hospital NHS Trust; both of Manchester, United Kingdom.
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
 -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 35SO4
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.
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