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. 2019 Sep 30;129(11):4739–4744. doi: 10.1172/JCI128205

Figure 2. Treatment with Pip6a-PMO normalizes global transcriptome at both expression and splicing levels.

Figure 2

Transcriptomic analysis by RNA sequencing was performed on total RNA isolated from gastrocnemius muscles of treated HSA-LR mice compared with HSA-LR and WT mice (n = 3). (A and B) Principal component analysis (A) and heatmap graphic (B) of all significantly expressed transcripts (adj. P < 0.1) reveal a global correction of the gene expression profile with Pip6a-PMO treatment. (C) Global correction of gene expression by Pip6a-PMO treatment (n = 376; FC ≥ 2, adj. P < 0.1): 85.5% of transcripts return to FC < 2 with an average correction index of 76%; 8% of transcripts remain at FC ≥ 2 but with correction index > 20%; 6.5% of transcripts are not corrected. (D) Heatmap graphic of all significant deregulated exon_bin (normalized counts) reveals a global correction of missplicing events with Pip6a-PMO treatment. (E) Overall correction of alternative splicing profiles by Pip6a-PMO treatment (n = 339 splicing events; FC ≥ 2, adj. P < 0.1): 80% of events return to FC < 2 with an average correction index of 83%; 11% remain at FC ≥ 2 but with correction index > 20%; 8% are not corrected.