Figure 8.
Chemically stabilized siRNA survives for weeks in acidic intracellular compartments. An Advanced ESC GalNAc–siRNA (siTTR-2) targeting Ttr was dosed SC at 0.5 mg/kg (all cohorts including the control group, which is shown in each graph as a comparison). GalNAc-INF7 endolytic peptide was dosed SC at 5 mg/kg in cohorts of mice at either 8 h (A) or days 11 (B), 14 (C) or 21 (D) post siRNA dose. Serum TTR protein levels were monitored through day 45. Data is represented as mean ± SD, for N = 3. Welch's independent t-test was utilized to compare the mean value between the control and peptide-treated groups at each timepoint to identify significant differences in target knockdown(*P ≤ 0.05, **P ≤ 0.01). Full results for the statistical analysis are included in the Supplemental Information.