Figure 4.

ASOs Reduce the Number of RNA-Foci-Positive Cells in SCA36 iPSCs

(A) Schema of NOP56 pre-mRNA structure and the ASO target (gold bars). (B) Representative images of RNA FISH analysis in SCA36 iPSCs treated with five kinds of ASOs. Scale bar, 10 μm. (C) The graph shows the number of RNA-foci-positive cells. The mean number of RNA-foci-positive cells in the control ASO treatment group was standardized as 1.0. Compared to the control ASO, all five ENA ASOs significantly reduced the percentage of RNA-foci-positive cells in SCA36 patient iPSCs. ***p < 0.001. Data are presented as mean ± SEM from n = 3 clones. (D) The graph shows the NOP56 mRNA expression levels in healthy control and SCA36 patient iPSCs. The mean NOP56 mRNA expression level of control iPSCs was standardized to be 1.0. NOP56 mRNA expression levels were significantly lower in SCA36 patient iPSCs than in control iPSCs. *p < 0.05. Data are presented as mean ± SEM from n = 3 clones. (E) The graph shows the NOP56 mRNA expression levels in SCA36 patient iPSCs after ASO treatment. The mean NOP56 mRNA expression level of the control ASO treatment group was standardized as 1.0. Compared to the control ASO, NOP56 mRNA expression levels were significantly decreased by ASO-B, ASO-C, ASO-D, and ASO-E but maintained by ASO-A. *p < 0.05, **p < 0.01. Values are expressed as mean ± SEM from n = 3 clones.