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

Abstract

	•In vivo application of a therapeutic gene editing strategy for permanent deletion of the pathogenetic CTG-repeat amplification in the DMPK gene that causes myotonic dystrophy type 1.
	•Following treatment, diseased mice show a significant improvement of both molecular and phenotypic defects.

Keywords

CRISPR/Cas9 / CTG repeats / DM1 / DMPK / DMSXL mouse model / gene editing / muscle / MyoAAV / myotonic dystrophy type 1

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