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https://hdl.handle.net/2440/59970
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Type: | Journal article |
Title: | SGSH gene transfer in mucopolysaccharidosis type IIIA mice using canine adenovirus vectors |
Author: | Lau, A. Hopwood, J. Kremer, E. Hemsley, K. |
Citation: | Molecular Genetics and Metabolism, 2010; 100(2):168-175 |
Publisher: | Academic Press Inc Elsevier Science |
Issue Date: | 2010 |
ISSN: | 1096-7192 1096-7206 |
Statement of Responsibility: | Adeline A. Lau, John J. Hopwood, Eric J. Kremer, and Kim M. Hemsley |
Abstract: | Many viral backbones have been used as gene transfer vectors. However, the efficacy of therapy based on human-derived vectors may be limited by the high incidence of pre-existing humoral and cellular memory immunity. To circumvent some of the clinical disadvantages of vectors derived from common human pathogens, we have used an E1-deleted vector derived from a xenogenic adenovirus, canine adenovirus serotype 2 (CAV-2) to ameliorate neuropathological changes associated with the lysosomal storage disorder, mucopolysaccharidosis type IIIA (MPS IIIA). This presently untreatable condition is caused by N-sulfoglucosamine sulfohydrolase (SGSH) deficiency and is characterized by heparan sulfate accumulation and progressive neurodegeneration. Injection of CAV-SGSH-GFP into the thalamus of adult MPS IIIA mouse brain resulted in short-term gene expression. In contrast, intra-ventricular injection of newborn mice yielded dose-dependent transgene expression which persisted for at least 20-weeks and improved neuropathology. Together, these studies suggest that this E1-deleted CAV-2 vector is capable of mediating regional medium-term gene expression and facilitating improvements in neuropathology in MPS IIIA mice. |
Keywords: | Mucopolysaccharidosis Sanfilippo syndrome Canine adenovirus Gene therapy |
Rights: | Copyright © 2010 Elsevier Inc. All rights reserved. |
DOI: | 10.1016/j.ymgme.2010.02.006 |
Published version: | http://dx.doi.org/10.1016/j.ymgme.2010.02.006 |
Appears in Collections: | Aurora harvest 5 Paediatrics publications |
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