Restore Cardiac Dystrophin Expression with Exon Skipping

In the vast majority of DMD patients, mutations disrupt the open reading frame (Koenig et al. 1989; Monaco et al. 1988). Restoring the reading frame by exon skipping holds great promise in ameliorating muscle disease (van Deutekom et al. 2007). Despite body-wide correction in the skeletal muscle, traditional exon skipping strategy has thus far had minimal success in the heart (Alter et al. 2006; Yokota et al. 2009). This hurdle is now resolved with modified antisense oligonu-cleotides (AON). In one approach, investigators conjugated AON with cell penetrating peptide to achieve efficient cardiac correction (Jearawiriyapaisarn et al. 2008; Wu et al. 2008; Yin et al. 2008a; b). In another approach, nonpeptide polymers, such as an octa-guanidine-conjugated morpholino AON, were found to result in widespread dystrophin restoration in the heart (Wu et al. 2009).

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