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rdf:type |
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lifeskim:mentions |
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pubmed:issue |
4
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pubmed:dateCreated |
2008-7-22
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pubmed:abstractText |
Alpha-sarcoglycan (alpha-SG) deficiency (limb-girdle muscular dystrophy [LGMD] type 2D) is the most common form of sarcoglycan-LGMD. No treatment is currently available. Prior studies suggest that overexpression of alpha-SG via adeno-associated virus (AAV)-mediated gene transfer results in poorly sustained gene expression related to transgene toxicity. These findings potentially preclude gene therapy as a treatment approach for LGMD2D.
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pubmed:grant |
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pubmed:commentsCorrections |
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pubmed:language |
eng
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pubmed:journal |
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pubmed:citationSubset |
AIM
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pubmed:chemical |
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pubmed:status |
MEDLINE
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pubmed:month |
Jul
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pubmed:issn |
1526-632X
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pubmed:author |
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pubmed:issnType |
Electronic
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pubmed:day |
22
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pubmed:volume |
71
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pubmed:owner |
NLM
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pubmed:authorsComplete |
Y
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pubmed:pagination |
240-7
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pubmed:dateRevised |
2008-11-21
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pubmed:meshHeading |
pubmed-meshheading:18525034-Animals,
pubmed-meshheading:18525034-Clinical Trials as Topic,
pubmed-meshheading:18525034-Creatine Kinase,
pubmed-meshheading:18525034-Cytomegalovirus,
pubmed-meshheading:18525034-Dependovirus,
pubmed-meshheading:18525034-Gene Expression,
pubmed-meshheading:18525034-Gene Therapy,
pubmed-meshheading:18525034-Gene Transfer Techniques,
pubmed-meshheading:18525034-Genetic Vectors,
pubmed-meshheading:18525034-Humans,
pubmed-meshheading:18525034-Mice,
pubmed-meshheading:18525034-Mice, Inbred C57BL,
pubmed-meshheading:18525034-Mice, Knockout,
pubmed-meshheading:18525034-Muscle, Skeletal,
pubmed-meshheading:18525034-Muscular Dystrophies, Limb-Girdle,
pubmed-meshheading:18525034-Myositis,
pubmed-meshheading:18525034-Promoter Regions, Genetic,
pubmed-meshheading:18525034-Sarcoglycans,
pubmed-meshheading:18525034-Treatment Outcome,
pubmed-meshheading:18525034-Up-Regulation
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pubmed:year |
2008
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pubmed:articleTitle |
Lack of toxicity of alpha-sarcoglycan overexpression supports clinical gene transfer trial in LGMD2D.
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pubmed:affiliation |
Center for Gene Therapy, Ohio State University, USA.
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pubmed:publicationType |
Journal Article,
Research Support, N.I.H., Extramural
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