title: miR-31 modulates dystrophin expression: new implications for Duchenne muscular dystrophy therapy.
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.
authors.affiliation: Department of Biology and Biotechnology C. Darwin, Institut Pasteur Cenci-Bolognetti and IBPM-Sapienza, University of Rome, Piazzele Aldo Moro 5, Rome 00185, Italy.
authors.forename: Davide
authors.initials: D
authors.lastname: Cacchiarelli
authors.forename: Tania
authors.initials: T
authors.lastname: Incitti
authors.forename: Julie
authors.initials: J
authors.lastname: Martone
authors.forename: Marcella
authors.initials: M
authors.lastname: Cesana
authors.forename: Valentina
authors.initials: V
authors.lastname: Cazzella
authors.forename: Tiziana
authors.initials: T
authors.lastname: Santini
authors.forename: Olga
authors.initials: O
authors.lastname: Sthandier
authors.forename: Irene
authors.initials: I
authors.lastname: Bozzoni
mesh_terms: D020413:3' Untranslated Regions; D000818:Animals; D002454:Cell Differentiation; D002478:Cells, Cultured; D016189:Dystrophin / Q000096:biosynthesis* / Q000235:genetics; D020869:Gene Expression Profiling; D006801:Humans; D051379:Mice; D018101:Mice, Inbred mdx; D035683:MicroRNAs / Q000096:biosynthesis*; D020388:Muscular Dystrophy, Duchenne / Q000378:metabolism* / Q000473:pathology / Q000628:therapy; D009224:Myotonia Congenita / Q000378:metabolism / Q000473:pathology; D034622:RNA Interference; D012333:RNA, Messenger / Q000378:metabolism; D032496:Satellite Cells, Skeletal Muscle / Q000166:cytology / Q000378:metabolism
chemical_list: D020413:3' Untranslated Regions; D016189:Dystrophin; C534612:MIRN31 microRNA, human; D035683:MicroRNAs; D012333:RNA, Messenger