Correction of Sanfilippo a Skin Fibroblasts By Retroviral Vector-Mediated Gene Transfer

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dc.contributor.authorBielicki, J.
dc.contributor.authorHopwood, J.
dc.contributor.authorAnson, D.
dc.date.issued1996
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.identifier.citationHuman Gene Therapy, 1996; 7(16):1965-1970
dc.identifier.doi10.1089/hum.1996.7.16-1965
dc.identifier.issn1043-0342
dc.identifier.issn1557-7422
dc.identifier.urihttp://hdl.handle.net/2440/7324
dc.language.isoen
dc.publisherMARY ANN LIEBERT INC PUBL
dc.source.urihttps://doi.org/10.1089/hum.1996.7.16-1965
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
pubs.publication-statusPublished

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