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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.

SUBMITTER: Cacchiarelli D

PROVIDER: S-EPMC3049433 | biostudies-literature | 2011 Feb

REPOSITORIES: biostudies-literature

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

Cacchiarelli Davide D Incitti Tania T Martone Julie J Cesana Marcella M Cazzella Valentina V Santini Tiziana T Sthandier Olga O Bozzoni Irene I

EMBO reports 20110107 2

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 inhibitio ...[more]

PMID: 21212803

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