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Comparison of the selectivity of an ASO and 2. (A) Precise recognition of r(CCUG)exp by 2 compared to ASO-treated cells. Effect on RNAs containing r(CCUG) repeats as determined by RTqPCR; WT CNBP is that derived from a non-DM2 or healthy fibroblast that does not express r(CCUG)exp. (B) ASO RNA-targeting strategy. (C) Small molecule RNA-targeting strategy. Error bars represent SD. **P < 0.01, ***P < 0.001, ****P < 0.0001 as determined by a one-way ANOVA (n = 3).
