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Article

AAV-microdystrophin Therapy Improves Cardiac Performance in Aged Female mdx Mice

2 Aug 2011

Abstract excerpt

Dystrophin deficiency leads to lethal dilated Duchenne cardiomyopathy. A promising therapy is to deliver a highly abbreviated microdystrophin gene to the heart using adeno-associated virus (AAV). Microdystrophin has been shown to mitigate dystrophin-deficient skeletal muscle disease. However, it is not clear whether microdystrophin is equally effective in treating Duchenne cardiomyopathy. To evaluate...

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PMID: 21811246
DOI: 10.1038/mt.2011.154

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