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

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

Abstract 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) o...

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Main Authors: Mariapaola Izzo, Jonathan Battistini, Elisabetta Golini, Christine Voellenkle, Claudia Provenzano, Tiziana Orsini, Georgios Strimpakos, Ferdinando Scavizzi, Marcello Raspa, Denisa Baci, Svetlana Frolova, Spyros Tastsoglou, Germana Zaccagnini, Jose Manuel Garcia‐Manteiga, Genevieve Gourdon, Silvia Mandillo, Beatrice Cardinali, Fabio Martelli, Germana Falcone
Format: Article
Language:English
Published: Wiley 2025-02-01
Series:Clinical and Translational Medicine
Subjects:
CRISPR/Cas9
CTG repeats
DM1
DMPK
DMSXL mouse model
gene editing
Online Access:https://doi.org/10.1002/ctm2.70227
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https://doi.org/10.1002/ctm2.70227

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