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.
2011
gene therapy; dmd; myoblasts; dystrophin; mirna
01 Pubblicazione su rivista::01a Articolo in rivista
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]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11573/377206
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