Extended Data Fig. 3: Scn2a-CRISPRa off-target analysis in cell lines. | Nature

Extended Data Fig. 3: Scn2a-CRISPRa off-target analysis in cell lines.

From: CRISPR activation for SCN2A-related neurodevelopmental disorders

Extended Data Fig. 3: Scn2a-CRISPRa off-target analysis in cell lines.

a. RNA-seq expression levels of Scn2a TAD-domain genes from Scn2a-rAAV-CRISPRa treated Neuro-2a cells compared to VP64-only. Circles are individual replicates, box plots are medians, quartiles and 100% tails. p-values noted above data. Wald test. b. RNA-seq expression levels of sodium channels from Scn2a-rAAV-CRISPRa treated Neuro-2a cells compared to VP64-only. Circles are individual replicates, box plots are medians, quartiles and 100% tails. p-values noted above data. Wald test. c. Volcano plot representing Log2 fold-change in expression levels for each gene. Wald test. P-values noted above each comparison. Grey dots represent no significant DEGs for CRISPRa, purple dots signify Scn2a and nearby genes, and orange dots denote upregulated genes. d. Gene ontology (GO) analysis showing enrichment of molecular functions. e. Gene ontology (GO) analysis showing enrichment of biological processes. f. Analysis of sgRNA sequence off-targeting using Cas-OFFinder.

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