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.
MiR-31 modulates dystrophin expression: New implications for Duchenne muscular dystrophy therapy / Cacchiarelli, Davide; Incitti, Tania; Martone, Julie; Cesana, Marcella; Cazzella, Valentina; Santini, Tiziana; Sthandier, Olga Elena; Bozzoni, Irene. - In: EMBO REPORTS. - ISSN 1469-221X. - STAMPA. - 12:2(2011), pp. 136-141. [10.1038/embor.2010.208]
MiR-31 modulates dystrophin expression: New implications for Duchenne muscular dystrophy therapy
CACCHIARELLI, DAVIDE;INCITTI, TANIA;MARTONE, Julie;CESANA, MARCELLA;CAZZELLA, VALENTINA;SANTINI, Tiziana;STHANDIER, Olga Elena;BOZZONI, Irene
2011
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' 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.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.