DMD and Duchenne muscular dystrophy: Oligonucleotide-based exon skipping is a powerful method to exclude specific exons and has been exploited to restore dystrophin expression by removing exons adjacent to genomic deletions and restoring the normal reading frame.18 This strategy has predominantly been used to skip exon 51, which can address up to 13% of all DMD patient deletions.19,20 However, this transient restoration requires regular administration of the exon skipping drug for the duration of treatment.