DMD and Duchenne muscular dystrophy: Among them, antisense oligonucleotide (AO)-mediated exon-skipping has been demonstrated as a promising therapy to treat Duchenne muscular dystrophy (DMD) by facilitating ‘skipping’ of specific dystrophin gene exon(s) to restore the reading frame of the mutated transcripts (Hoffman et al., 1987; Koenig et al., 1989; Amantana et al., 2007; van Deutekom et al., 2007; Wu et al., 2008, 2009, 2010, 2012; Yin et al., 2008; Kinali et al., 2009; Goemans et al., 2011; Mendell et al., 2013).