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https://hdl.handle.net/2440/7324
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DC Field | Value | Language |
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dc.contributor.author | Bielicki, J. | - |
dc.contributor.author | Hopwood, J. | - |
dc.contributor.author | Anson, D. | - |
dc.date.issued | 1996 | - |
dc.identifier.citation | Human Gene Therapy, 1996; 7(16):1965-1970 | - |
dc.identifier.issn | 1043-0342 | - |
dc.identifier.issn | 1557-7422 | - |
dc.identifier.uri | http://hdl.handle.net/2440/7324 | - |
dc.description.abstract | The 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.iso | en | - |
dc.publisher | MARY ANN LIEBERT INC PUBL | - |
dc.source.uri | http://dx.doi.org/10.1089/hum.1996.7.16-1965 | - |
dc.subject | Cell Line | - |
dc.subject | 3T3 Cells | - |
dc.subject | Fibroblasts | - |
dc.subject | Skin | - |
dc.subject | Animals | - |
dc.subject | Mice | - |
dc.subject | Moloney murine leukemia virus | - |
dc.subject | Mucopolysaccharidosis III | - |
dc.subject | Hydrolases | - |
dc.subject | Glycosaminoglycans | - |
dc.subject | Transfection | - |
dc.subject | Gene Transfer, Horizontal | - |
dc.subject | Genetic Vectors | - |
dc.title | Correction of Sanfilippo a Skin Fibroblasts By Retroviral Vector-Mediated Gene Transfer | - |
dc.type | Journal article | - |
dc.identifier.doi | 10.1089/hum.1996.7.16-1965 | - |
pubs.publication-status | Published | - |
Appears in Collections: | Aurora harvest 5 Paediatrics publications |
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