Figure 4.

UROS Exon 10 Subclone Genotyping

(A) Sanger sequence of six porphyrin-negative subclones. The corrected mutation is highlighted in red and the silent restriction site ApaI in yellow. HDR and indel analysis by RFLP and ICE are indicated.

(B) UROS enzymatic activity of polyclonal porphyrin-negative sorted population and corrected subclones. Data are represented as mean ± SEM, ^∗∗∗p < 0.001 versus CEP-hIPSC lines, n ≥ 4 independent experiments for CEP and polyclonal porphyrin-negative (corrected) cell lines. n = 6 corrected subclones.