Skip to main content
Fig. 5 | Genome Biology

Fig. 5

From: Antisense suppression of the nonsense mediated decay factor Upf3b as a potential treatment for diseases caused by nonsense mutations

Fig. 5

ASO-mediated Upf3b depletion stabilizes hFIX-R29X mRNA in hemophilia mice. hFIX-R29X mice aged 8–14 weeks (n = 5–6, 2–3 female and 2–3 male mice per group) were treated every five days with six total doses of DPBS, Control-GalNAc-ASO (15 mg/kg), Upf1-GalNAc-ASO (10 mg/kg), or Upf3b-GalNAc-ASO (10 mg/kg). Animals were sacrificed 48 h after the last dose. Untreated FIX knockout mice (KO) and hFIX-WT mice were used as controls. Results are presented as means ± standard errors. ad mRNA levels of the indicated NMD factors that are also endogenous NMD substrates were analyzed by qPCR from mouse liver total RNA samples. Gapdh was used as an endogenous control. The expression levels in DPBS-treated mouse liver were set as 1. e hFIX mRNA levels measured by qPCR. Gapdh was used as an endogenous control. hFIX mRNA level in hFIX-WT mouse liver was set as 1. f Mouse plasma hFIX protein levels as measure by ELISA. Statistical significance was determined using a one-way ANOVA and Dunnett’s multiple comparison test in Prism. All groups were compared to DPBS-treated hFIX-R29X group. ** p < 0.01; *** p < 0.001; **** p < 0.0001

Back to article page