Please use this identifier to cite or link to this item:
https://hdl.handle.net/2440/7324
Citations | ||
Scopus | Web of ScienceĀ® | Altmetric |
---|---|---|
?
|
?
|
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 |
Publisher: | MARY ANN LIEBERT INC PUBL |
Issue Date: | 1996 |
ISSN: | 1043-0342 1557-7422 |
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 Fibroblasts Skin Animals Mice Moloney murine leukemia virus Mucopolysaccharidosis III Hydrolases Glycosaminoglycans Transfection Gene Transfer, Horizontal Genetic Vectors |
DOI: | 10.1089/hum.1996.7.16-1965 |
Published version: | http://dx.doi.org/10.1089/hum.1996.7.16-1965 |
Appears in Collections: | Aurora harvest 5 Paediatrics publications |
Files in This Item:
There are no files associated with this item.
Items in DSpace are protected by copyright, with all rights reserved, unless otherwise indicated.