期刊
EMBO REPORTS
卷 12, 期 2, 页码 136-141出版社
WILEY
DOI: 10.1038/embor.2010.208
关键词
DMD; dystrophin; miRNA; myoblasts; gene therapy
资金
- Microsoft
- Telethon [GGP07049]
- Parent Project Italia
- EU [LSHG-CT-2006-037900]
- ESF
- IIT
- PRIN
- BEMM
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 30 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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