Development of a Clinical AAV-Based Vector Encoding a Microdystrophin Transgene for the Treatment of...
Development of a Clinical AAV-Based Vector Encoding a Microdystrophin Transgene for the Treatment of Duchenne Muscular Dystrophy
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Full title
Development of a Clinical AAV-Based Vector Encoding a Microdystrophin Transgene for the Treatment of Duchenne Muscular Dystrophy
Author / Creator
Journal title
Molecular therapy, 2013-09, Vol.21 (9), p.e17-e17
Language
English
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Scope and Contents
Contents
Duchenne muscular dystrophy (DMD) is a recessively-inherited muscle wasting disorder caused by mutations within the dystrophin gene. Gene therapy using adeno-associated viral (AAV) vectors to deliver dystrophin expression cassettes remains a promising treatment option that would be applicable to all patients regardless of their underlying genetic m...
Alternative Titles
Full title
Development of a Clinical AAV-Based Vector Encoding a Microdystrophin Transgene for the Treatment of Duchenne Muscular Dystrophy
Authors, Artists and Contributors
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Record Identifier
TN_cdi_proquest_miscellaneous_1673399635
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_1673399635
Other Identifiers
ISSN
1525-0016
E-ISSN
1525-0024
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