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 3-2 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.
Original language | English (US) |
---|---|
Pages (from-to) | 136-141 |
Number of pages | 6 |
Journal | EMBO Reports |
Volume | 12 |
Issue number | 2 |
DOIs | |
State | Published - Feb 2011 |
Externally published | Yes |
Keywords
- DMD
- dystrophin
- gene therapy
- miRNA
- myoblasts