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rdf:type |
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lifeskim:mentions |
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pubmed:issue |
1
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pubmed:dateCreated |
2004-1-12
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pubmed:abstractText |
Fabry disease is a recessive, X-linked disorder caused by a deficiency of the lysosomal enzyme alpha-galactosidase A, leading to an accumulation of the glycosphingolipid globotriaosylceramide (GL-3) in most tissues of the body. The goal of this study was to determine if systemic delivery of a nonviral vector could correct the enzyme deficiency and reduce the levels of GL-3 in different tissues of a transgenic knockout mouse model of the disease.
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pubmed:language |
eng
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pubmed:journal |
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pubmed:citationSubset |
IM
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pubmed:chemical |
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pubmed:status |
MEDLINE
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pubmed:month |
Jan
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pubmed:issn |
1099-498X
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pubmed:author |
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pubmed:copyrightInfo |
Copyright 2003 John Wiley & Sons, Ltd.
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pubmed:issnType |
Print
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pubmed:volume |
6
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pubmed:owner |
NLM
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pubmed:authorsComplete |
Y
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pubmed:pagination |
85-92
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pubmed:dateRevised |
2006-11-15
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pubmed:meshHeading |
pubmed-meshheading:14716680-Animals,
pubmed-meshheading:14716680-Disease Models, Animal,
pubmed-meshheading:14716680-Fabry Disease,
pubmed-meshheading:14716680-Female,
pubmed-meshheading:14716680-Gene Expression,
pubmed-meshheading:14716680-Gene Therapy,
pubmed-meshheading:14716680-Genetic Vectors,
pubmed-meshheading:14716680-Lipids,
pubmed-meshheading:14716680-Mice,
pubmed-meshheading:14716680-Mice, Inbred BALB C,
pubmed-meshheading:14716680-Plasmids,
pubmed-meshheading:14716680-Trihexosylceramides,
pubmed-meshheading:14716680-alpha-Galactosidase
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pubmed:year |
2004
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pubmed:articleTitle |
Partial correction of the alpha-galactosidase A deficiency and reduction of glycolipid storage in Fabry mice using synthetic vectors.
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pubmed:affiliation |
Genzyme Corporation, 31 New York Avenue, Framingham, MA 01701-9322, USA.
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pubmed:publicationType |
Journal Article
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