MiR-31 modulates dystrophin expression: New implications for Duchenne muscular dystrophy therapy

Davide Cacchiarelli, Tania Incitti, Julie Martone, Marcella Cesana, Valentina Cazzella, Tiziana Santini, Olga Sthandier, Irene Bozzoni

Research output: Contribution to journalArticlepeer-review

98 Scopus citations

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 languageEnglish (US)
Pages (from-to)136-141
Number of pages6
JournalEMBO Reports
Volume12
Issue number2
DOIs
StatePublished - Feb 1 2011

Keywords

  • DMD
  • dystrophin
  • gene therapy
  • miRNA
  • myoblasts

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