| 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. |