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. 2022 Aug 14;26:547–561. doi: 10.1016/j.omtm.2022.08.005

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© 2022 The Authors

This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).

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On-target gene specificity supported by RNA-seq analysis

To increase the sensitivity and the power to detect any small but significantly altered genes, we combined 10 targeted clonal lines targeted by L4-R4 and 10 clonal lines targeted by L4-R6 gRNA combinations to be compared with 12 EV-treated controls. (A) Significance values (uncorrected p values on the y axis) were compared with log2 (fold-change) (x axis) to highlight significantly altered genes. A horizontal and a vertical line represent Bonferroni-corrected significance and zero fold-change, respectively. (B) Expression levels of total HTT (mutant plus normal) based on the DGE analysis are summarized in a boxplot. Total HTT levels were decreased by 38% in TP-CRISPR targeted clonal lines.