Figure 3.

Combination of erythromycin and pafuramidine rescues multiple mis-splicing events and reduces HSA transgene levels in HSA^LR DM1 mice. PSI between WT and HSA^LR mice with and without treatment determined via RT-PCR. Mice were treated with either 600 mg kg^–1 erythromycin (EM, green), 10 mg kg^–1 pafuramidine (PaF, blue) alone, or a combination of both (EM+PaF, magenta) per oral administration daily for 14 days. (A) Atp2a1 exon22, (B) Clcn1 exon7a, (C) Clasp1 exon13, and (D) Nfix exon7 mis-splicing events all displayed additive rescue with combination treatment. Mean % rescue ± standard deviation values are displayed below each graph. Expression levels in HSA^LR mice with and without treatment determined via RT-qPCR. PaF (blue) treatment alone and the combination (magenta) reduced (E) HSA transgene levels and did not affect (F) endogenous Dmpk levels, while EM (green) treatment alone did not significantly reduce either. Mean relative expression ± standard deviation values are displayed below each graph.