Fig. 6: Exon activation in SMA patient cells by CASFx. | Nature Communications

Fig. 6: Exon activation in SMA patient cells by CASFx.

From: CRISPR artificial splicing factors

Fig. 6

a Schematic of the two all-in-one CASFx constructs (CASFx-1: RBFOX1N-dCasRx-C and RBFOX1N-dPspCas13b-C) and six gRNAs targeting the corresponding numbered locations on the endogenous intron. b Upper panel shows inclusion/exclusion ratio fold-changes of the endogenous SMN2 gene in patient cells assayed by qRT-PCR 5 days after nucleofection with the indicated CASFx and gRNA relative to 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 using primers En-F and En-R on endogenous SMN2 transcripts in the indicated samples. Uncropped gel images and qRT-PCR values are included in the Source Data file.

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