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Systemic <i>Trans</i> -Splicing Adeno-Associated Viral Delivery Efficiently Transduces the Heart of Adult <i>mdx</i> Mouse, a Model for Duchenne Muscular Dystrophy

23 Jul 2009

Abstract excerpt

Trans-splicing adeno-associated viral (tsAAV) vectors hold great promise for delivering large therapeutic genes. One potential application is in the treatment of Duchenne muscular dystrophy (DMD). In this case, it is necessary to transduce whole body muscle. We demonstrated body-wide AAV-9 tsAAV transduction in normal neonatal mice. However, it was not clear whether such an approach would work in diseased mice....

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PMID: 19627234
DOI: 10.1089/hum.2009.058

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