Fig. 8: Relationship of ACTA1-CUGexp transgene and myosin gene expression in treated muscles.

a We used ddPCR to quantify expression of human ACTA1-CUGexp mRNA, human ACTA1-CUGexp pre-mRNA, and reference gene mouse Acta1 in TA muscles of LR41;Mbnl1−/− (N = 10) treated with Clcn1 ASO (+) or invert oligo (−) as copies/µl cDNA. Untreated WT TA muscles (−) (N = 4) served as controls. b Using the copies/µl cDNA data in a), we normalized expression of ACTA1-CUGexp mRNA (left) and ACTA1-CUGexp pre-mRNA (right) to mouse Acta1 in LR41;Mbnl1−/− (N = 10). **P < 0.01; *P < 0.05 (one-way ANOVA). c ddPCR quantification of Dmpk expression in treated muscles as copies/µl cDNA (left) and normalized to Acta1 (right) n LR41;Mbnl1−/− (N = 10) and WT (N = 4). d Relationship of % Myh2 (left), Myh1 (middle), and Myh4 (right) gene expression (x-axes) with ACTA1-CUGexp mRNA normalized to Acta1 and e with ACTA1-CUGexp pre-mRNA (y-axes) in LR41;Mbnl1−/− TA muscles (N = 10) treated for 16–18 days (black circles) or 52 days (orange triangles). The Pearson correlation coefficient r and P-value for each are shown. Error bars indicate ±s.e.m. Source data are provided as a Source Data file.