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Type: Journal article
Title: Correction of Sanfilippo a Skin Fibroblasts By Retroviral Vector-Mediated Gene Transfer
Author: Bielicki, J.
Hopwood, J.
Anson, D.
Citation: Human Gene Therapy, 1996; 7(16):1965-1970
Issue Date: 1996
ISSN: 1043-0342
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.
Keywords: Cell Line
3T3 Cells
Moloney murine leukemia virus
Mucopolysaccharidosis III
Gene Transfer, Horizontal
Genetic Vectors
DOI: 10.1089/hum.1996.7.16-1965
Appears in Collections:Aurora harvest 5
Paediatrics publications

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