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Int J Mol Sci. 2018 Dec 18;19(12). pii: E4104. doi: 10.3390/ijms19124104.

Dissecting Pathogenetic Mechanisms and Therapeutic Strategies in Drosophila Models of Myotonic Dystrophy Type 1.

Author information

1
GReD, INSERM U1103, CNRS, UMR6293, University of Clermont Auvergne, 28 Place Henri Dunant, 63000 Clermont-Ferrand, France. anissa.souidi@uca.fr.
2
GReD, INSERM U1103, CNRS, UMR6293, University of Clermont Auvergne, 28 Place Henri Dunant, 63000 Clermont-Ferrand, France. monika.zmojdzian@uca.fr.
3
GReD, INSERM U1103, CNRS, UMR6293, University of Clermont Auvergne, 28 Place Henri Dunant, 63000 Clermont-Ferrand, France. christophe.jagla@uca.fr.

Abstract

Myotonic dystrophy type 1 (DM1), the most common cause of adult-onset muscular dystrophy, is autosomal dominant, multisystemic disease with characteristic symptoms including myotonia, heart defects, cataracts and testicular atrophy. DM1 disease is being successfully modelled in Drosophila allowing to identify and validate new pathogenic mechanisms and potential therapeutic strategies. Here we provide an overview of insights gained from fruit fly DM1 models, either: (i) fundamental with particular focus on newly identified gene deregulations and their link with DM1 symptoms; or (ii) applied via genetic modifiers and drug screens to identify promising therapeutic targets.

KEYWORDS:

DM1; Drosophila; animal model; drug screen; genetic screen; muscular dystrophy; therapeutic targets

PMID:
30567354
PMCID:
PMC6321436
DOI:
10.3390/ijms19124104
[Indexed for MEDLINE]
Free PMC Article

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