The most promising therapy to date for the severely debilitating neuromuscular disorder DMD is treatment with AOs, which restores the reading frame of the dystrophin pre-mRNA by exon skipping. Two AOs, a PMO 10,11 and a 2′OMe oligonucleotide, 8, 9 are currently in clinical trials and the early Ten micrograms of total protein was loaded (TA, quadriceps, gastrocnemius, diaphragm, heart, and abdomen muscles) relative to 50% (5 µg protein) and 10% (1 µg) C57BL10 controls, and normalized to α-actinin loading control (for quantification see Supplementary Figure S2a) . PMO, phosphorodiamidate morpholino oligonucleotide.