2 ome ps

Previously, four AONs were designed targeting the top SC35 motifs and the mutation itself [18 (link)]. For the detailed screening that was the subject of this study, the entire pseudoexon plus the flanking regions were analyzed for their RNA structure to identify the open and closed regions. Subsequently, AONs were designed according to previously described guidelines independently of the potential motifs that they were targeting [35 (link),36 (link)]. All AON sequences and properties are provided in Table 1. After AON design, targeted regions were analyzed to predict potential exonic splicing enhancer (ESE) motifs using either an ESE finder (http://krainer01.cshl.edu/cgi-bin/tools/ESE3/esefinder.cgi?process=home), which allows for the detection of SRSF1, SRSF2, SRSF5, and SRSF6, or using RBPmap (http://rbpmap.technion.ac.il/index.html), which allows for the identification of 94 potential binding sites for RNA binding proteins. All AONs were 2’OMe-PS (2’O-methyl phosphorothioate) and were purchased from Eurogentec (Liege, Belgium). Sequences and general parameters of the 26 AONs and 2 SONs are depicted in Table 1.