Figure 2 | Scientific Reports

Figure 2

From: Spinal muscular atrophy phenotype is ameliorated in human motor neurons by SMN increase via different novel RNA therapeutic approaches

Figure 2

MO-10-34 increases SMN in SMA iPSCs.

(A) Schematic representation of the PMO-10-34 sequence and its interaction with the ISS-N1 target of SMN2 pre-mRNA. (B) After treating iPSCs with PMO-10-34, immunocytochemical analysis revealed a greater representation of nuclear gems (green) in MO-treated SMA iPSCs than in scrambled SMA-treated iPSCs. Nuclei are labeled with DAPI (blue). Western blot (C) and quantitative RT-PCR (D) analyses demonstrated a significant >2-fold increase in the expression of full-length SMN protein in treated SMA iPSCs compared with scrambled SMA-treated iPSCs (*p < 0.01; ANOVA). Values represent means ± s.e.m. from five independent experiments performed in triplicate. (E) Gems quantification confirmed the increase in the number of gems in MO-treated vs. scramble-treated cells. Gems counts in wild-type (WT) and heterozygous (HET) cells are shown for comparison (*p < 0.01, ANOVA). Scale bar: 75 μm. Values represent means ± s.e.m. from five independent experiments performed in triplicate.

Back to article page