Fig. 4

Patient-derived hiPSC editing using pAIO-PE2-GFP and pAIO-PE4max. A Comparison of the editing efficiency when correcting the heterozygous KCNQ2 R201C mutation using CMV-PE2-GFP and CMV-PE3-GFP versus pAIO-PE2-GFP and pAIO-PE4max. B Comparison of the editing efficiency when correcting heterozygous KCNQ2 R201C mutation using pegRNA-C201R (only correction KCNQ2 R201C) versus pegRNA-C201R-P (correction KCNQ2 R201C + introduction PAM-removal mutation) using pAIO-PE2-GFP and pAIO-PE4max. C FACS showing the two GFP-positive fractions that were separated when sorting the GFP populations. D Editing efficiency of pAIO-PE2-GFP after sorting on the high and low GFP expressing fractions using pegRNA-C201R and pegRNA-C201R-P. E Number of clones that carry the R201C correction using the (left) pegRNA-C201R and (right) pegRNA-C201R-P. The target region and design of pegRNA-C201R and pegRNA-C201R-P can be found in Additional file 1: Fig. S9. F Miseq data of pAIO-PE2-GFP- pegRNA-C201R-P high GFP population showing that the PAM-removal mutation (black arrow) is only present in reads that do not carry the KCNQ2-R201C mutation (red arrow). Bar plots show the mean edit percentages ± SD, replicates are presented as individual data points (see individual data values in Additional file 2: Table S1)