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. 2017 Oct 2;7:12532. doi: 10.1038/s41598-017-12844-z

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© The Author(s) 2017

Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons license and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/.

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Activity of selected gapmers, designed to SNCA RNA 46 and RNA 53 targets, in HeLa cells. (A) Results of ASOs transfection to HeLa cells. The EC₅₀ values for gapmers tested in presence of WT and mutant alleles were estimated based on dose-response curves fitted to experimental data in Origin Lab 8.0 software (for statistics see Supplementary data). The data were gathered from at least three separate experiments involving five concentrations in the range 10–100nM. Comparison of EC₅₀ values for gapmers between WT and Mutant target RNA alleles allowed to evaluate allele-preference for cleavage of tested gapmers. Two last columns of the table present WT/Mut ratio of gapmer selectivity and the selectivity of mismatched gapmers relative to complementary gapmers (kKM for RNA 46 target and hKM for RNA 53 target). (B) Dose-response curves for antisense gapmers causing preferential cleavage of Mut 46 RNA (kKM) and Mut 53 RNA (h2).