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Article

Dual exon skipping in myostatin and dystrophin for Duchenne muscular dystrophy

20 Apr 2011

Abstract excerpt

BACKGROUND: Myostatin is a potent muscle growth inhibitor that belongs to the Transforming Growth Factor-β (TGF-β) family. Mutations leading to non functional myostatin have been associated with hypermuscularity in several organisms. By contrast, Duchenne muscular dystrophy (DMD) is characterized by a loss of muscle fibers and impaired regeneration. In this study, we aim to knockdown myostatin by means of exon...

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PMID: 21507246
DOI: 10.1186/1755-8794-4-36

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