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

First Author  Cacchiarelli D Year  2011
Journal  EMBO Rep Volume  12
Issue  2 Pages  136-41
PubMed ID  21212803 Mgi Jnum  J:168844
Mgi Id  MGI:4939089 Doi  10.1038/embor.2010.208
Citation  Cacchiarelli D, et al. (2011) miR-31 modulates dystrophin expression: new implications for Duchenne muscular dystrophy therapy. EMBO Rep 12(2):136-41
abstractText  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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