,2 This so-called exon-skipping strategy can be achieved using antisense oligonucleotides (ASOs) that interfere with splicing signals or regulating elements in the exon, thus leading to the skipping of the targeted exon at the precursor (pre-)mRNA level.3, 4, 5, 6 The applicability of ASO-based therapies has now been demonstrated in clinical trials using different chemistries of ASOs targeting the human dystrophin exon 51 in DMD patients. Here, DMD is linked to Duchenne muscular dystrophy.