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. Author manuscript; available in PMC: 2017 Oct 16.
Published in final edited form as: Nature. 2017 Apr 12;544(7650):367–371. doi: 10.1038/nature22038

Figure 4. ASOs targeting ataxin 2 extend lifespan and improve motor performance in TDP-43 transgenic mice.

Figure 4

a) P1 mouse pups were treated with Ctrl or Atxn2 ASO via ICV injection. At P21, mRNA levels of Atxn2 (b) were decreased by 77% in the Atxn2 ASO-injected mouse brains without affecting mRNA levels of the human TDP-43 transgene (c). d) Lifespan was significantly extended by Atxn2 ASO treatment (n = 16) vs Ctrl ASO (n = 17). Curves were compared by log rank test, and effect size was estimated using a Cox proportional hazards model. e) Gait impairment score was also improved in Atxn2 ASO treated mice (n = 14) by P21 compared to Ctrl ASO (n = 20). Two-tailed t-tests were used to compare the two treatment groups at each age. Means are indicated, and error bars indicate S.E.M. *p < 0.05