Please use this identifier to cite or link to this item: https://hdl.handle.net/2440/7324
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dc.contributor.authorBielicki, J.-
dc.contributor.authorHopwood, J.-
dc.contributor.authorAnson, D.-
dc.date.issued1996-
dc.identifier.citationHuman Gene Therapy, 1996; 7(16):1965-1970-
dc.identifier.issn1043-0342-
dc.identifier.issn1557-7422-
dc.identifier.urihttp://hdl.handle.net/2440/7324-
dc.description.abstractThe recent cloning of the sulfamidase gene has made possible the consideration of gene-based therapies for Sanfilippo A syndrome (mucopolysaccharidosis type IIIA), one of the most common of the mucopolysaccharidoses. In this paper, we present the construction of a retroviral vector in which a sulfamidase cDNA is under the transcriptional control of the Moloney murine leukemia virus long terminal repeat. This construct was used to make a high-titer (4 x 10(5) colony-forming units/ml) producer cell line, PA317/LNSSN#19, in the amphotropic packaging cell line PA317. This producer cell line was shown to be helper virus free using an assay for horizontal spread of virus. Virus supernatant from PA317/LNSSN#19 was used to transduce Sanfilippo A fibroblasts, resulting in complete correction of both the enzymatic defect and the storage phenotype as assessed by intracellular accumulation of 35SO4(-)-labeled material. Phenotypic correction was seen even when the levels of viral transduction were low. These results show that gene therapy of the Sanfilippo A syndrome is practicable, although the nature of the disorder suggests that careful consideration needs to be given to the choice of the cellular target for gene transfer.-
dc.language.isoen-
dc.publisherMARY ANN LIEBERT INC PUBL-
dc.subjectCell Line-
dc.subject3T3 Cells-
dc.subjectFibroblasts-
dc.subjectSkin-
dc.subjectAnimals-
dc.subjectMice-
dc.subjectMoloney murine leukemia virus-
dc.subjectMucopolysaccharidosis III-
dc.subjectHydrolases-
dc.subjectGlycosaminoglycans-
dc.subjectTransfection-
dc.subjectGene Transfer, Horizontal-
dc.subjectGenetic Vectors-
dc.titleCorrection of Sanfilippo a Skin Fibroblasts By Retroviral Vector-Mediated Gene Transfer-
dc.typeJournal article-
dc.identifier.doi10.1089/hum.1996.7.16-1965-
pubs.publication-statusPublished-
Appears in Collections:Aurora harvest 5
Paediatrics publications

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