Figure 3. Exon 53 skipping and dystrophin.
(A) Treatment with golodirsen demonstrates an increase in skipping of exon 53 in dystrophin mRNA. Percent exon 53 skipping for individual patients at baseline (open circles) and on treatment (filled squares). Data represent mean ± SD values for 4–8 replicates. (B) Positive correlation between percent exon 53 skipping and production of dystrophin protein. Percent exon 53 skipping change from baseline was plotted against the change in dystrophin protein from baseline, as measured by Western blot (Spearman r correlation coefficient: 0.500; p = 0.011).