Figure 4.

Antisense oligonucleotides at a variety of concentrations induce skipping of PE50.1 in patient iFDM cells. (A) Using experimental conditions in Figure 3, iFDM cells (duplicate cultures for each treatment) from patients JF196, TDM57 and a normal (NHDF‐2) control were treated with AON2 or scrambled (SCR) control AONs at the concentrations shown or TE alone. In both patient cell lines, AON2 treatment at each concentration tested reduced PE50.1 mutant mRNA expression compared with SCR or TE controls. (B) Quantitative RT‐PCR analysis of the RNAs in panel A shows that treatment of patient iFDMs with AON2 significantly reduces the expression of the mutant transcripts compared to SCR and TE controls (*P < 0.05) (gray bars, mutant transcripts; black bars, normal transcripts; mean ± SD). The relative expression of each form is calculated using the amplification of a PCR product spanning the exon 13‐14 junction as representative of 100% DYSF expression.