Fig. 2

Correction of the NIPBL c.5483G > A substitution in hiPSCs derived from a CdLS patient. A Editing efficiencies analyzed by TIDER in hiPSCs-c.5483G > A electroporated with SpHiFiCas9-gRNA + 4 RNPs and ssODN-CdLS untreated (−) or treated ( +) with NU7441. B HDR/InDels ratio analyzed in cells treated as in A. C Sanger sequences of hiPSCs-c.5483G > A on the left and a fully corrected clone (hiPSCs-cl1) on the right. The red arrow indicates the target edit and blue arrows indicate the substitutions introduced in the ssODN to avoid Cas9 re-cleavage. Data were obtained from n ≥ 3 experiments. Data are means ± SD. Statistical analysis was performed using two-way ANOVA; *P ≤ 0.05, **P ≤ 0.01, ***P ≤ 0.001