Figure 4.

Gene-edited and patient-derived homozygous c.5461-10T>C ROs show high levels of rescued ABCA4 transcript upon AON treatment

(A) Transcript analysis of gene edited ROs treated with a 1.5 μM 2′MOE-modified and 2′OMe-modified AONs for 28 days. (Top) The 4-week-long treatment used a wash-out regimen in which the AON was added only at day 0 of treatment and its concentration was halved by each medium change every other day. (B) The percentage of splicing correction in patient-derived ROs treated with QR-1011. For the 2 × 10 μM group, QR-1011 was administered at treatment day 0 and 14. A dose-dependent increase in splice correction was observed from 21.2% to 53.4%. Data are shown as mean ± standard error of the mean, n = 6 per condition. Asterisks display the significant differences vs. scrambled oligo or untreated (∗p ≤ 0.05, ∗∗p ≤ 0.01, ∗∗∗p ≤ 0.001, ∗∗∗∗p ≤ 0.0001).