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

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Cacchiarelli, Davide | Incitti, Tania | Martone, Julie | Cesana, Marcella | Cazzella, Valentina | Santini, Tiziana | Sthandier, Olga | Bozzoni, Irene

Edité par CCSD ; EMBO Press -

International audience. 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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