Abstract
Antisense-mediated exon-skipping is a potential treatment for the lethal muscle wasting condition Duchenne muscular dystrophy (DMD). Local treatment in the dystrophic mdx mouse model leads to efficient restoration of dystrophin expression. Antisense can also be delivered systemically but requires repeated administration and high doses to achieve a therapeutically useful effect. The article by Jearawiriyapaisarn and colleagues1 in this issue of Molecular Therapy addresses the problem of low efficiency of systemic delivery by coupling an arginine-rich cell-penetrating peptide (CPP) to a phosphorodiamidate morpholino oligomer (PMO).
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