| SGSH gene transfer in mucopolysaccharidosis type IIIA mice using canine adenovirus vectors. | |
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MedLine Citation:
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PMID: 20231109 Owner: NLM Status: MEDLINE |
Abstract/OtherAbstract:
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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. |
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Authors:
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Adeline A Lau; John J Hopwood; Eric J Kremer; Kim M Hemsley |
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Publication Detail:
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Type: Journal Article; Research Support, Non-U.S. Gov't Date: 2010-02-14 |
Journal Detail:
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Title: Molecular genetics and metabolism Volume: 100 ISSN: 1096-7206 ISO Abbreviation: Mol. Genet. Metab. Publication Date: 2010 Jun |
Date Detail:
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Created Date: 2010-05-17 Completed Date: 2010-08-09 Revised Date: - |
Medline Journal Info:
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Nlm Unique ID: 9805456 Medline TA: Mol Genet Metab Country: United States |
Other Details:
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Languages: eng Pagination: 168-75 Citation Subset: IM |
Affiliation:
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Lysosomal Diseases Research Unit, SA Pathology at the WCH, North Adelaide, Australia. adeline.lau@adelaide.edu.au |
Export Citation:
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| MeSH Terms | |
Descriptor/Qualifier:
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Adenoviruses, Canine
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genetics*,
immunology Animals Animals, Newborn Antibodies, Viral / analysis Enzyme Replacement Therapy / methods* Gene Transfer Techniques Genetic Vectors Hydrolases / therapeutic use* Mice Mucopolysaccharidosis III / genetics, therapy* |
| Chemical | |
Reg. No./Substance:
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0/Antibodies, Viral; EC 3.-/Hydrolases; EC 3.10.1.1/N-sulfoglucosamine sulfohydrolase |
From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine
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