Furthermore, gapmer-mediated Ptbp1 depletion (Supplementary Fig. 4a) or ASO disruption of PTBP binding (Supplementary Fig. 4b) improved SYNGAP1 productive splicing in immortalized neuroblastoma cells (mouse N2a, human SH-SY5Y), in which the basal inclusion of Exon 11 AS is high (Supplementary Fig. 4a, b). The gene discussed is SYNGAP1; the disease is neuroblastoma.