Nature Communications (Apr 2023)
Correction of Clcn1 alternative splicing reverses muscle fiber type transition in mice with myotonic dystrophy
Abstract
In a double homozygous mouse model of myotonic dystrophy type 1, Hu et al. use antisense oligonucleotide correction of myotonia to induce a therapeutic shift from an overabundance of oxidative muscle fibers to mechanically stronger glycolytic fibers.