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. 2018 May 29;8:8234. doi: 10.1038/s41598-018-26481-7

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© The Author(s) 2018

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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Effect of PTC124 treatment on CHM3 iPSC-derived RPE. (A) qPCR analysis of CHM mRNA levels in PTC124-treated patient iPSC-derived RPE (dotted bars). CHM transcript levels in non-treated (NT) and DMSO-treated CHM3 RPE (grey bars) are significantly lower than those of wild-type (WT; black bars) cells. Data are expressed as mean ± SEM, n = 3; Mann and Whitney test *p < 0.05. PTC124 treatment of CHM3 iPSC-derived RPE, regardless of the concentration used, did not significantly alter CHM transcript levels as compared to DMSO-treated cells; Kruskal-Wallis test p > 0.05. (B) A representative in vitro prenylation assay showing biotinylated Rab proteins in wild-type (WT), and in non-treated (NT), DMSO-treated or PTC124-treated CHM3 iPSC-derived RPE. β-actin serves as a loading control. Bands were cropped from the same gel. (C) Quantification of the relative biotinylated Rab levels in multiple independent assays, after normalisation to β-actin loading, shows a significant 4-fold increase in the non-treated (NT) or DMSO-treated iPSC-derived RPE of patient CHM3 (grey bars), as compared to wild-type RPE (WT; black bar). Data are expressed as mean ± SEM; n is indicated in the corresponding histogram bars; Mann and Whitney test *p < 0.05. Treatment with PTC124 results in a non-significant decrease in biotinylated Rab levels at all doses (dotted bars), with the exception of 100 µg/ml, as compared to DMSO-treated cells (red line); Kruskal-Wallis test p > 0.05.