Skip to main content
. Author manuscript; available in PMC: 2021 Sep 23.
Published in final edited form as: Neuron. 2020 Jul 15;107(6):1124–1140.e11. doi: 10.1016/j.neuron.2020.06.027

Figure 3: Sense repeat RNA targeting ASOs mitigate alterations in specific Nups, restore the localization of Ran GTPase, and protect against cellular toxicity in C9orf72 iPSNs.

Figure 3:

(A-E) Maximum intensity projections from SIM imaging of Nups in nuclei isolated from 5 μM sense strand targeting or scrambled ASO treated control and C9orf72 iPSNs. Treatment as indicated on left, genotype and antibodies as indicated on top. (F-T) Quantification of Nup spots and volume. N = 5 control and 5 C9orf72 iPSC lines, 50 NeuN+ nuclei per line/treatment. Two-way ANOVA with Tukey’s multiple comparison test was used to calculate statistical significance. **** p < 0.0001. (U) Confocal imaging of 5 μM sense strand targeting or scrambled ASO treated control and C9orf72 iPSNs immunostained for Ran. Treatment as indicated on left, genotype and antibodies as indicated on top. (V) Quantification of nuclear to cytoplasmic ratio of Ran. N = 3 control and 3 C9orf72 iPSC lines, 30 cells per line. Two-way ANOVA with Tukey’s multiple comparison test was used to calculate statistical significance. **** p < 0.0001. (W) Quantification of percent cell death following exposure to glutamate. N = 4 control and 4 C9orf72 iPSC lines, 10 frames per well. Two-way ANOVA with Tukey’s multiple comparison test was used to calculate statistical significance. **** p < 0.0001. Scale bar = 5 μm (A-E), 10 μm (U). * indicates significantly altered Nups, ** indicates significantly restored Nups.