Fig. 5
From: Stargardt disease-associated in-frame ABCA4 exon 17 skipping results in significant ABCA4 function
AON-based treatment achieved significant levels of ABCA4 Δexon 17 transcripts. A The percentage of ABCA4 mRNA lacking exon 17 in WT WERI-Rb-1 cells after 48 h long AON treatments, n = 3. B The percentage of ABCA4 mRNA lacking exon 17 in WT retinal organoids is significantly increased upon exposure to the most effective AONs in a 4-week long wash-out treatment, n = 6. The data is shown as mean ± SEM. Asterisks display the significant differences vs. untreated or scrambled AON (*p ≤ 0.05, **p ≤ 0.01, ***p ≤ 0.001, ****p ≤ 0.0001)