Figure 3.

Morphological and transcript comparison of wild-type and gene-edited ROs

(A) Morphology assessed at day 120 of organoid differentiation suggests ROs derived from homozygous c.5461-10T>C and the control isogenic parent cell line displayed neural retina (thin light rim at the margin of the ROs) and the brush border with photoreceptor cells. (B) Analyses of the total transcript expression of CRX, NRL, NR2E3, USH2A, and ABCA4 (see Table S3). CRX and the photoreceptor markers were expressed similarly in both groups. ABCA4 was expressed at higher levels in wild-type organoids. Data are shown as mean ± standard error of the mean, n = 4. (C) Comparison of ABCA4 transcript expression in c.5461-10T>C and wild-type ROs and midigenes showed that the relative levels of the three transcripts are comparable between RO and midigene for both WT and the c.5461-10T>C variant. Data are shown as mean ± standard error of the mean, ∗∗∗p ≤ 0.0001.