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dc.contributor.authorAnson, D.-
dc.contributor.authorMcIntyre, C.-
dc.contributor.authorThomas, B.-
dc.contributor.authorKoldej, R.-
dc.contributor.authorRanieri, E.-
dc.contributor.authorDerrick Roberts, A.-
dc.contributor.authorClements, P.-
dc.contributor.authorDunning, K.-
dc.contributor.authorByers, S.-
dc.identifier.citationGenetic Vaccines and Therapy, 2007; 5(6):WWW 1-WWW 8-
dc.descriptionThe electronic version of this article is the complete one and can be found online at:
dc.description.abstractBackground Mucopolysaccharidosis type IIIA (MPS IIIA) is the most common of the mucopolysaccharidoses. The disease is caused by a deficiency of the lysosomal enzyme sulphamidase and results in the storage of the glycosaminoglycan (GAG), heparan sulphate. MPS IIIA is characterised by widespread storage and urinary excretion of heparan sulphate, and a progressive and eventually profound neurological course. Gene therapy is one of the few avenues of treatment that hold promise of a sustainable treatment for this disorder. Methods The murine sulphamidase gene cDNA was cloned into a lentiviral vector and high-titre virus produced. Human MPS IIIA fibroblast cultures were transduced with the sulphamidase vector and analysed using molecular, enzymatic and metabolic assays. High-titre virus was intravenously injected into six 5-week old MPS IIIA mice. Three of these mice were pre-treated with hyperosmotic mannitol. The weight of animals was monitored and GAG content in urine samples was analysed by polyacrylamide gel electrophoresis. Results Transduction of cultured MPS IIIA fibroblasts with the sulphamidase gene corrected both the enzymatic and metabolic defects. Sulphamidase secreted by gene-corrected cells was able to cross correct untransduced MPS IIIA cells. Urinary GAG was found to be greatly reduced in samples from mice receiving the vector compared to untreated MPS IIIA controls. In addition, the weight of treated mice became progressively normalised over the 6-months post-treatment. Conclusion Lentiviral vectors appear promising vehicles for the development of gene therapy for MPS IIIA.-
dc.description.statementofresponsibilityDonald S Anson, Chantelle McIntyre, Belinda Thomas, Rachel Koldej, Enzo Ranieri, Ainslie Roberts, Peter R Clements, Kylie Dunning and Sharon Byers-
dc.publisherBioMed Central Ltd.-
dc.rights© 2007 Anson et al; licensee BioMed Central Ltd. This is an Open Access article distributed under the terms of the Creative Commons Attribution License (, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.-
dc.titleLentiviral-mediated gene correction of mucopolysaccharidosis type IIIA-
dc.typeJournal article-
dc.identifier.orcidDunning, K. [0000-0002-0462-6479]-
dc.identifier.orcidByers, S. [0000-0001-5576-3636]-
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Obstetrics and Gynaecology publications

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