Figure 2

Concentration series of exon 44 and exon 45 AONs. At a concentration of 100 nM (100) and up, levels of wild type transcript increased for the exon 45 duplicated patient (upper panel) compared to the untreated (NT) and 50 nM (50) treated samples. Double exon 45 skipping at low levels was observed for all samples. These levels increase slightly at 100 nM and higher but do not increase further at higher concentrations. For the exon 44 duplicated patient (lower panel), variable low levels of wild type transcripts are present in each sample with the regular exon 44 AON and a short suboptimal AON (short), but as dystrophin was not restored this probably reflects fluctuating levels of the wild type-like artefact. At concentrations that are generally effective (100 nM and up), the levels of original transcript decrease and the amount of double exon 44 skipped transcript increases. M is 100 bp DNA marker, HC is healthy control.