Fig. 4

Dystrophin PTC-containing mRNA is stabilized in mdx mice treated with Upf3b- and Smg6-ASOs. Male mdx mice (aged five weeks; n = 4) were treated with DPBS, Control ASO (100 mg/kg/week), Upf1-ASO (50 mg/kg/week), Smg6-ASO (100 mg/kg/week), or Upf3b-ASO (100 mg/kg/week) for five weeks. Animals were sacrificed 48 h after the last ASO dose. Total RNA was isolated from mouse TA muscle. mRNA levels of NMD factors and dystrophin were analyzed by qPCR and normalized to total RNA as measured using Ribogreen. The expression levels in DPBS-treated mouse TA muscle were set as 1. Results are presented as means ± standard errors. a Upf3b mRNA levels. b Smg6 mRNA levels. c Upf1 mRNA levels. d Dystrophin mRNA levels. Dystrophin mRNA level in wild-type DPBS treated mice was included as control. Statistical significance was determined using a one-way ANOVA and Dunnett’s multiple comparison test in Prism. All groups were compared to DPBS-treated mdx mouse group. * p < 0.05; ** p < 0.01; *** p < 0.001; **** p < 0.0001