Muscle-specific gene editing improves molecular and phenotypic defects in a mouse model of myotonic dystrophy type 1.

doi:10.1002/ctm2.70227

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Article

Muscle-specific gene editing improves molecular and phenotypic defects in a mouse model of myotonic dystrophy type 1.

Clinical and translational medicine - 1 Feb 2025

Izzo Mariapaola, Battistini Jonathan, Golini Elisabetta, Voellenkle Christine, Provenzano Claudia, Orsini Tiziana, Strimpakos Georgios, Scavizzi Ferdinando, Raspa Marcello, Baci Denisa, Frolova Svetlana, Tastsoglou Spyros, Zaccagnini Germana, Garcia-Manteiga Jose Manuel, Gourdon Genevieve, Mandillo Silvia, Cardinali Beatrice, Martelli Fabio, Falcone Germana

Abstract excerpt

BACKGROUND: Myotonic dystrophy type 1 (DM1) is a genetic multisystemic disease, characterised by pleiotropic symptoms that exhibit notable variability in severity, nature and age of onset. The genetic cause of DM1 is the expansion of unstable CTG-repeats in the 3' untranslated region (UTR) of the DMPK gene, resulting in the accumulation of toxic CUG-transcripts that sequester RNA-binding proteins and form nuclear...

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PMID: 39956955
DOI: 10.1002/ctm2.70227
PMCID: PMC11830570

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DMPK

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