Figure 4.

Diamidine analogs show variable mis-splicing rescue in DM2 and DM1 fibroblasts

(A–H) RT-PCR splicing analysis of the common alternative exon events INSR exon 11, FLNB exon 31, and SYNE1 exon65 in DM1 (blue) and DM2 (green) fibroblasts treated for 4 days with either group one compounds, DB1242 (A) and DB1210 (B); group two compounds, DB829 (C), DB1246 (D), DB1247 (E), and DB1273 (F); and group three compounds, DB270 (G) and Compound 7 (Cmpd7) (H). All compound groups show variable mis-splicing rescue across DM1 and DM2 fibroblasts. Structures of the diamidine analogs are shown on the top right corner of each graph. All experiments were done in at least triplicate using biological replicates (two-tailed t-test ∗∗p < 0.01, ∗∗∗p < 0.001).