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miR-31 modulates dystrophin expression: new implications for Duchenne muscular dystrophy therapy.

Abstract : Duchenne muscular dystrophy (DMD)--which is caused by mutations in the dystrophin gene-is one of the most severe myopathies. Among therapeutic strategies, exon skipping allows the rescue of dystrophin synthesis through the production of a shorter but functional messenger RNA. Here, we report the identification of a microRNA--miR-31--that represses dystrophin expression by targeting its 3' untranslated region. In human DMD myoblasts treated with exon skipping, we demonstrate that miR-31 inhibition increases dystrophin rescue. These results indicate that interfering with miR-31 activity can provide an ameliorating strategy for those DMD therapies that are aimed at efficiently recovering dystrophin synthesis.
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Submitted on : Thursday, April 10, 2014 - 12:29:13 PM
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Davide Cacchiarelli, Tania Incitti, Julie Martone, Marcella Cesana, Valentina Cazzella, et al.. miR-31 modulates dystrophin expression: new implications for Duchenne muscular dystrophy therapy.. EMBO Reports, EMBO Press, 2011, 12 (2), pp.136-41. ⟨10.1038/embor.2010.208⟩. ⟨pasteur-00976757⟩

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