Antisense Modulation of Both Exonic and Intronic Splicing Motifs Induces Skipping of a DMD Pseudo-Exon Responsible for X-Linked Dilated Cardiomyopathy

Antisense-mediated exon skipping has been actively investigated as a treatment strategy for subsets of Duchenne muscular dystrophy mutations. This approach is based on targeting specific splicing motifs that interfere with the spliceosome assembly resulting in an in-frame deletion of a deleterious mutation. In this study, Rimessi et al. use a variety of in vitro strategies to examine the role of intronic splicing sequences with the goal of designing improved exon skipping-mediated therapeutic approaches.