Fig. 1.

Lipidoid formulation and effects of PCSK9 silencing in wild-type mice. (A) Cationic lipidoid component structure of the formulation. (B) Dose-dependent decrease in hepatic PCSK9 mRNA (relative to controls) 2 days after dose (n = 6 per group). (C) Liver PCSK9 mRNA and total serum cholesterol levels in mice (n = 5 per group) 3 days after a dose of 5 mg/kg LNP-PCS-A2 or PBS. (D) Duration of hepatic PCSK9 transcript silencing in mice (n = 5 per group) after a single injection of 5 mg/kg or 7.5 mg/kg LNP-PCS-A2. (B) one-way ANOVA with Student's t test; (C and D) two-way ANOVA with Bonferroni test. (B–D) Each value is the group mean ± STDEV. Asterisks represent statistical difference between PBS and PCSK9 siRNA treated groups. **, P ≤ 0.01; ***, P ≤ 0.001