Figure 2.

AON-mediated skipping of constitutive exons fails to reduce DMPK RNA. (A) Quantitation of total DMPK mRNA by qPCR (real-time PCR) expression analyses of the 3′UTR fragment demonstrates lack of significant DMPK transcript reduction upon e5 skipping in control and DM1-affected fibroblasts. DMPK expression is shown relative to GAPDH, increasing doses of AON targeting e5 (AON-4), control scrambled sequence AON (scr-1) are indicated. (B) Representative results of western blot analyses showing DMPK protein level in control fibroblasts dosed with 75 nM of indicated AONs. Three lanes indicate individual samples. Quantitation is shown to the right. Whole-membrane scan of DMPK western blot is shown in Supplementary Figure S4C.