Fig. 4

Dystrophin evaluation of the edited clone myotubes. (A) Representative confocal images of dystrophin immunofluorescence (green) in differentiated myotubes from: Control (C2), DMD, Edited∆45–55 and Im∆45–55-D1. Nuclei were stained with DAPI (blue) (scale bar = 50 μm). (B) Quantification and representative blots of dystrophin in protein extracts from 3 healthy controls (C1=▴, C2=▪, C3=♦); DMD, the Edited∆45–55 and Im∆45–55-D1 myotubes. Dystrophin levels (Dys1) were normalized to α-actinin signal (α-actn) (n = 4 technical replicates). (C) In-cell western quantification of dystrophin expression of three healthy controls (C1, C2, and C3), DMD, the Edited∆45–55 and Im∆45–55-D1. Dystrophin signal is normalised to cell number signal (Cell Tag) and set to 1 (mean of the three controls) (n = 6 wells). *p < 0.05, ***p < 0.001, ****p < 0.0001 according to Mann–Whitney U (B) and Kruskal-Wallis (C) and error bars represent the mean ± SEM