Modeling Myotonic Dystrophy Type 2 Using Drosophila melanogaster.
Int J Mol Sci
; 24(18)2023 Sep 16.
Article
em En
| MEDLINE
| ID: mdl-37762484
ABSTRACT
Myotonic dystrophy 2 (DM2) is a genetic multi-systemic disease primarily affecting skeletal muscle. It is caused by CCTGn expansion in intron 1 of the CNBP gene, which encodes a zinc finger protein. DM2 disease has been successfully modeled in Drosophila melanogaster, allowing the identification and validation of new pathogenic mechanisms and potential therapeutic strategies. Here, we describe the principal tools used in Drosophila to study and dissect molecular pathways related to muscular dystrophies and summarize the main findings in DM2 pathogenesis based on DM2 Drosophila models. We also illustrate how Drosophila may be successfully used to generate a tractable animal model to identify novel genes able to affect and/or modify the pathogenic pathway and to discover new potential drugs.
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Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Proteínas de Drosophila
/
Distrofia Miotônica
Tipo de estudo:
Prognostic_studies
Limite:
Animals
Idioma:
En
Ano de publicação:
2023
Tipo de documento:
Article