Figure 1.

Combination of furamidine and erythromycin displays an additive rescue of mis-splicing in DM1 patient-derived myotubes. Absolute value of the percent spliced-in difference between non-DM control and DM1 myotubes (|ΔPSI|) with and without treatment determined via RT-PCR. Cells were treated with either furamidine (FM) alone or erythromycin (EM) or a combination of both. (A) MBNL1 exon5, (B) MBNL2 exon5, (C) NUMA1 exon2, and (D) SYNE1 exon137 events all displayed additive mis-splicing rescue after 4 days of treatment with the combination. Treatment concentrations of 0.5 μM FM alone, 25 μM EM alone, and the combination at those concentrations with the mean % rescue ± standard deviation above are denoted with arrows for each event.