Figure 1.

CRISPR-Edited iPSC PRPF31^+/−

(A) Schematic representation of the PRPF31 locus. A 20-bp nucleotide gRNA sequence (blue line) is followed by PAM (red line) designed to target exon 7. Bottom sequence shows the 10-bp deletion found in clone no. 144, which was used for differentiation into RPE. (B) mRNA levels of PRPF31 normalized to GAPDH measured in triplicate, expressed by CRISPR-edited iPSC PRPF31^+/+ (wild-type [WT]) clones 156 and 157, and PRPF31^+/− (heterozygous [HET]) mutant clones 118 (4-bp deletion) and 144 (10-bp deletion). The average expression of WT cells was used as a value of 1 for relative quantification (two-way ANOVA, ****p < 0.0001; data are represented as mean ± SD).