Figure 4.

Risdiplam and branaplam promote off-target exon skipping. (A) Table summarizing splicing-relevant information regarding top exons, skipping of which was increased by HiR treatment only, by both HiR and HiB treatments and by HiB treatment only. Table contents are similar to those in Figure 3A. (B–D) Genomic views of RNA-Seq reads (upper panels) and RT–PCR results (lower panels) examining ESK events that are triggered by HiR only (B), both HiR and HiB (C) and HiB only (D) in GM03813 fibroblasts. For genomic views, red arrows indicate the location of the affected exon. Other labeling and coloring are the same as in Figure 3B–D.