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Article

Gene Therapy in Large Animal Models of Muscular Dystrophy

1 Jan 2009

Abstract excerpt

The muscular dystrophies are a group of genetically and phenotypically heterogeneously inherited diseases characterized by progressive muscle wasting, which can lead to premature death in severe forms such as Duchenne muscular dystrophy (DMD). In many cases they are caused by the absence of proteins that are critical components of the dystrophin-glycoprotein complex, which links the cytoskeleton and the basal...

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PMID: 19293461
DOI: 10.1093/ilar.50.2.187

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