Fig. 7
Gene correction of PRPF31 mutation results in reversal of cellular and functional phenotypes in RPE and photoreceptors. a–c CRISPR/Cas9 correction of the PRPF31 deletion in exon 11; d, e Quantification of cilia length and incidence in PRPF31- and WT-RPE. f TEM analysis of PRPF31-edited RPE cilia showing morphologically normal cilia, scale bar: 500 nm; g Increased phagocytosis in PRPF31-edited RPE. h–j Restoration of apical–basal polarity in PRPF31-edited RPE, scale bar: 50 μm. k, l Quantification of cilia length and frequency in PRPF31- and WT-photoreceptors; m TEM analysis of PRPF31-edited photoreceptor cilia showing morphologically normal cilia, scale bar: 500 nm. c–e, g–i, k, l Data shown as mean ± SEM, n = 3. Statistical significance of pairwise comparisons is indicated by n.s.: not significant; *p < 0.05; **p < 0.01; ***p < 0.001; ****p < 0.0001 (Student’s paired t test)
