Fig. 2: Activation and repression of exon by differential positioning of CASFx.

a Schematic of the CRISPR artificial splicing factors, CASFx-1 (RBFOX1N-dCasRx-C), CASFx-2 (RBM38-dCasRx), CASFx-3 (dCasRx-RBM38) and SMN2 minigene, as well as a set of three target sites downstream of E7 (DN: gRNA-1 through 3) and one target site target within E7 (EX). b Upper panel shows the inclusion/exclusion (inc/exc) ratio fold-change assayed by qRT-PCR on SMN2 minigene transcripts in cells co-transfected with dCasRx, CASFx, and the indicated gRNAs, with respect to the GFP control (set to 1). Data are represented as mean ± SD (n = 3). Lower panel shows a gel image of semi-quantitative splicing RT-PCR of the corresponding samples. “C” indicates a control gRNA without matching SMN2 minigene sequence; “DN” indicates a pool of three gRNAs (SMN2-gRNA-1 through 3) targeting downstream of E7; “EX” indicates a gRNA targeting within E7. Uncropped gel images and qRT-PCR values are included in the Source Data file.