Fully Deleted Adenovirus Persistently Expressing GAA Accomplishes Long-Term Skeletal Muscle Glycogen...
Fully Deleted Adenovirus Persistently Expressing GAA Accomplishes Long-Term Skeletal Muscle Glycogen Correction in Tolerant and Nontolerant GSD-II Mice
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Publisher
United States: Elsevier Limited
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Language
English
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Publisher
United States: Elsevier Limited
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Contents
Glycogen storage disease type II (GSD-II) patients manifest symptoms of muscular dystrophy secondary to abnormal glycogen storage in cardiac and skeletal muscles. For GSD-II, we hypothesized that a fully deleted adenovirus (FDAd) vector expressing hGAA via nonviral regulatory elements (PEPCK promoter/ApoE enhancer) would facilitate long-term effica...
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Full title
Fully Deleted Adenovirus Persistently Expressing GAA Accomplishes Long-Term Skeletal Muscle Glycogen Correction in Tolerant and Nontolerant GSD-II Mice
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TN_cdi_proquest_miscellaneous_67575215
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_67575215
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ISSN
1525-0016
E-ISSN
1525-0024
DOI
10.1016/j.ymthe.2005.08.006
