Blood
Department of Haematology, Royal Manchester Children's Hospital, United Kingdom.
Published: March 2002
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.
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http://dx.doi.org/10.1182/blood.v99.5.1857 | DOI Listing |
Adv Sci (Weinh)
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Department of Orthopaedics, The First Affiliated Hospital of Soochow University, Orthopedic Institute, MOE Key Laboratory of Geriatric Diseases and Immunology, Suzhou Medical College, Soochow University, Suzhou, Jiangsu, 215000, China.
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Department of Immunology and Regenerative Biology, Weizmann Institute of Science, Rehovot, Israel.
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Trinity Centre for Biomedical Engineering, Trinity Biomedical Sciences Institute, Trinity College Dublin, Dublin, Ireland; Department of Mechanical, Manufacturing and Biomedical Engineering, School of Engineering, Trinity College Dublin, Dublin, Ireland; Department of Anatomy and Regenerative Medicine, Royal College of Surgeons in Ireland, Dublin, Ireland; Advanced Materials and Bioengineering Research Centre (AMBER), Royal College of Surgeons in Ireland and Trinity College Dublin, Dublin, Ireland. Electronic address:
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INRS- Centre Armand-Frappier Santé Biotechnologie, Université du Québec, Laval, QC, Canada.
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Department of Pediatrics, Shengjing Hospital of China Medical University, Shenyang, Liaoning, China.
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