Figure 4.

Sustainability of Dystrophin Protein Expression Induced by NS-065/NCNP-01

Western blot analysis of dystrophin protein expression in cells derived from patients with DMD involving deletion of (A) exons 45–52 and (B) exons 48–52 1 and 2 weeks after transfection with NS-065/NCNP-01, either once or twice over a period of 2 days. The positive control (P.C.) was a cell lysate of myotubes differentiated from normal human fibroblasts by transduction with MYOD. Densitometric analysis of the western blots of cells from patients with DMD involving deletion of (C) exons 45–52 and (D) exons 48–52 relative to each P.C. on the same membrane is shown. The bars show the mean and SD (n = 4). n.s., no significant difference; ***p < 0.001 (Tukey’s multiple comparison test). DMD, Duchenne muscular dystrophy.