Umfang:
Online-Ressource
ISSN:
1469-3178
Inhalt:
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.
Inhalt:
Exon skipping can rescue dystrophin synthesis in Duchenne muscular dystrophy. Here, miR‐31 is found to repress dystrophin translation such that miR‐31 depletion improves dystrophin production induced by exon skipping. Preventing miR‐31 activity might therefore increase the effectiveness of therapeutic attempts to enhance the levels of dystrophin.
In:
volume:12
In:
number:2
In:
year:2011
In:
pages:136-141
In:
extent:6
In:
European Molecular Biology Organization, EMBO reports, Heidelberg : EMBO Press, [200]-, 12, Heft 2 (2011), 136-141 (gesamt 6), 1469-3178
Sprache:
Englisch
DOI:
10.1038/embor.2010.208
URN:
urn:nbn:de:101:1-2023032004354970805754
URL:
https://doi.org/10.1038/embor.2010.208
URL:
https://nbn-resolving.org/urn:nbn:de:101:1-2023032004354970805754
URL:
https://d-nb.info/1283846217/34
URL:
https://doi.org/10.1038/embor.2010.208