miR-31 modulates dystrophin expression: new implications for Duchenne muscular dystrophy therapy

被引：120

作者：

Cacchiarelli, Davide

Incitti, Tania

Martone, Julie

Cesana, Marcella

Cazzella, Valentina

Santini, Tiziana

Sthandier, Olga

Bozzoni, Irene ^{[1
,2
]}

机构：

[1] Inst Pasteur Cenci Bolognetti, Dept Biol & Biotechnol C Darwin, I-00185 Rome, Italy

[2] Univ Rome, IBPM Sapienza, I-00185 Rome, Italy

来源：

EMBO REPORTS | 2011年 / 12卷 / 02期

关键词：

DMD; dystrophin; miRNA; myoblasts; gene therapy; GENE-THERAPY; MUSCLE-CELLS; MDX MOUSE; ANTISENSE; DIFFERENTIATION; MICE; RESTORATION; MODEL;

D O I：

10.1038/embor.2010.208

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

070307 [化学生物学]; 071010 [生物化学与分子生物学];

摘要：

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 30 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.

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页码：136 / 141

页数：6

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