Figure 8.

Silencing of Gys2 mRNA Expression Reduces Liver Toxicities in a L-G6pc^−/− GSD Ia Mouse Model

L-G6pc^−/− mice were dosed intravenously with PBS or GYS2-2 weekly for 5 weeks. Liver and blood samples were collected 48 hr following the final dose. (A) Analysis of serum samples was performed to measure circulating liver enzymes. (B) Measurement of caspase-3/-7 activity was performed on liver extracts to detect apoptosis. Numbers (N) of male (M) versus female (F) animals in each group are indicated. Unpaired t test for statistical significance relative to PBS control group or wild-type animal (****p < 0.0001; ***p ≤ 0.001; **p ≤ 0.01; *p ≤ 0.05). Data are presented as mean ± SD.