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

Autor: Irene Bozzoni, Julie Martone, Davide Cacchiarelli, Valentina Cazzella, Tania Incitti, Marcella Cesana, Olga Sthandier, Tiziana Santini
Přispěvatelé: Cacchiarelli, Davide, Incitti, Tania, Martone, Julie, Cesana, Marcella, Cazzella, Valentina, Santini, Tiziana, Sthandier, Olga, Bozzoni, Irene, Department of Biology and Biotechnology 'Charles Darwin', Institut Pasteur, Fondation Cenci Bolognetti - Istituto Pasteur Italia, Fondazione Cenci Bolognetti, Réseau International des Instituts Pasteur (RIIP)-Réseau International des Instituts Pasteur (RIIP)-Università degli Studi di Roma 'La Sapienza' = Sapienza University [Rome], This work was partly supported by grants from Telethon (GGP07049), Parent Project Italia, EU project SIROCCO (LSHG CT-2006-037900), ESF project 'NuRNASu', IIT 'SEED' PRIN and BEMM.
Rok vydání: 2011
Předmět:
Duchenne muscular dystrophy
Genetic enhancement
gene therapy
dmd
myoblasts
dystrophin
mirna
Biochemistry
Dystrophin
Mice
0302 clinical medicine
RNA interference
MESH: Animals
3' Untranslated Regions
Cells
Cultured
0303 health sciences
biology
MESH: Mice
Inbred mdx
MESH: Satellite Cells
Skeletal Muscle
MicroRNA
Cell Differentiation
MESH: 3' Untranslated Regions
musculoskeletal system
3. Good health
mir-31
RNA Interference
MESH: Myotonia Congenita
MESH: Cells
Cultured
Human
MESH: Cell Differentiation
musculoskeletal diseases
congenital
hereditary
and neonatal diseases and abnormalities
Myotonia Congenita
Satellite Cells
Skeletal Muscle
3' Untranslated Region
MESH: RNA Interference
MESH: Gene Expression Profiling
03 medical and health sciences
MESH: Dystrophin
Genetic
DMD
Utrophin
MESH: Muscular Dystrophy
Duchenne
Genetics
medicine
Animals
Humans
[SDV.BBM]Life Sciences [q-bio]/Biochemistry
Molecular Biology
RNA
Messenger
MESH: Mice
Molecular Biology
MESH: RNA
Messenger
miRNA
030304 developmental biology
MESH: Humans
Animal
Three prime untranslated region
business.industry
Gene Expression Profiling
Scientific Reports
medicine.disease
Exon skipping
Muscular Dystrophy
Duchenne
MicroRNAs
Mice
Inbred mdx
Cancer research
biology.protein
myoblast
business
MESH: MicroRNAs
030217 neurology & neurosurgery
Zdroj: EMBO Reports
EMBO Reports, EMBO Press, 2011, 12 (2), pp.136-41. ⟨10.1038/embor.2010.208⟩
ISSN: 1469-3178
1469-221X
DOI: 10.1038/embor.2010.208
Popis: International audience; 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.
Databáze: OpenAIRE
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