Efficacy of the novel LNA/DNA mixmers to induce SMN2 exon 7 inclusion. 5 nM transfection of AONs #1-#5 induced SMN2 exon 7 inclusion at a significantly higher rate than the mock AON transfection. (a) RT-PCR of SMN2 and GAPDH in human SMA patient fibroblasts (GM03813). AONs #1–8 were transfected at 5 nM concentration. Top band: exon 7-included. Bottom band: exon 7-excluded. (b) Quantification of SMN2 exon 7 inclusion in the AON-treated SMA fibroblasts by RT-PCR. (c) Relative expression of full-length SMN2 to GAPDH measured by qPCR. The data were normalized to non-treated cells. Bars represent mean ± S.D. of three independent experiments. One-way ANOVA with Dunnett’s multiple comparison test. M: mock control AON, NT: non-treated, H: healthy cells, B: blank.