Figure 6.

Combination treatment displays synergistic mis-splicing rescue in HSA^LR DM1 mice. Splicing analysis of ES events determined via RNA-seq on HSA^LR mice treated with 600 mg kg^–1 erythromycin (EM, green) or 10 mg kg^–1 pafuramidine (PaF, blue) or a combination of both (EM+PaF, magenta) per oral administration daily for 14 days. (A) Scatter plot displaying the theoretical percent rescue based upon a purely additive effect by adding the percent rescues of erythromycin and pafuramidine alone (gray circles) and the actual percent rescue with the combination treatment (magenta squares) for the 142 ES events rescued by the combination treatment ordered by increasing theoretical percent rescue. Gray dotted lines mark 10–110% rescue. The arrows denote the (B) Tnnt3 and (C ) Tpd52l2 mis-splicing events which displayed synergistic rescue with combination treatment. Mean % rescue ± standard deviation values are displayed below each graph.