{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T13:37:39Z","timestamp":1790861859003,"version":"4.1.0"},"reference-count":21,"publisher":"American Society of Hematology","issue":"5","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2002,3,1]]},"abstract":"<jats:p>We have investigated the utility of bone marrow\u2013derived 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\u2013expressing retrovirus. Green fluorescent protein\u2013positive cells maintained their proliferative and differentiation capacity. Next we used a vector encoding \u03b1-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 35SO4sequestered 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.<\/jats:p>","DOI":"10.1182\/blood.v99.5.1857","type":"journal-article","created":{"date-parts":[[2002,7,27]],"date-time":"2002-07-27T12:46:40Z","timestamp":1027774000000},"page":"1857-1859","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":33,"title":["Retrovirally mediated correction of bone marrow\u2013derived mesenchymal stem cells from patients with mucopolysaccharidosis type I"],"prefix":"10.1182","volume":"99","author":[{"given":"Melissa A.","family":"Baxter","sequence":"first","affiliation":[{"name":"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."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robert F.","family":"Wynn","sequence":"additional","affiliation":[{"name":"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."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jonathan A.","family":"Deakin","sequence":"additional","affiliation":[{"name":"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."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ilaria","family":"Bellantuono","sequence":"additional","affiliation":[{"name":"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."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kirsten G.","family":"Edington","sequence":"additional","affiliation":[{"name":"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."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alan","family":"Cooper","sequence":"additional","affiliation":[{"name":"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."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Guy T. N.","family":"Besley","sequence":"additional","affiliation":[{"name":"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."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Heather J.","family":"Church","sequence":"additional","affiliation":[{"name":"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."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"J. 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