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

Davide Cacchiarelli (1) , Tania Incitti (1) , Julie Martone (1) , Marcella Cesana (1) , Valentina Cazzella (1) , Tiziana Santini (1) , Olga Sthandier (1) , Irene Bozzoni (1)

1 Department of Biology and Biotechnology "Charles Darwin"

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.

Domains: Biochemistry, Molecular Biology

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, 2011, 12 (2), pp.136-41. ⟨10.1038/embor.2010.208⟩. ⟨pasteur-00976757⟩