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. 2022 Jun 23;50(12):6656–6670. doi: 10.1093/nar/gkac539

Figure 1.

Figure 1.

Evaluation of GNA-modified siRNAs in mice. (A) Structures of (S)-GNA, 2′-OMe and 2′-F nucleosides. Impact of GNA substitution at the specified position on activity (left) and liver guide strand concentration measured by RT-qPCR (right) in mice (n = 3) 7 days post-dose for Ttr-targeting (B) or Hao1-targeting (C) GalNAc–siRNAs dosed subcutaneously at either 0.5 mg/kg (Ttr) or 1.0 mg/kg (Hao1). Statistically significant differences relative to the parent siRNA are shown in each graph. (D) Impact of GNA substitution on Ttr mRNA knockdown in mice (n = 3) after a single subcutaneous dose of 0.75 mg/kg D1 or D4 and guide strand concentration in whole liver and Ago2 over time from the same study.