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

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Academic year: 2020

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Figure

Fig. 1 Nonsense-mediated degradation of aThe mRNA level of each NMD factor in untreated (NT) MHT cells was set as 1.activity from the PTC-containingerrors (n = 3).MHT cells
Fig. 2 Nonsense-mediated degradation of endogenous NMD substrates is inhibited by ASOs targeting NMD factors
Fig. 3 ASO-mediated Upf3b depletion is well tolerated in normal mice. Mice (n = 4) were treated with an Upf3b-ASO at 50, 100, or 150 mg/kg/week.DPBS and a scrambled ASO dosed at 150 mg/kg/week were used as controls
Fig. 4 Dystrophin PTC-containing mRNA is stabilized in mdx mice treated withdusing a one-way ANOVA and Dunnett0.05; **week) for five weeks
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