Figure 1.
Robust correction of OTC deficiency and orotic aciduria in adult Spfash mice. Adult male Spfash mice were injected intraperitoneally with 5 × 1010 (n = 5), 1.5 × 1011 (n = 6), or 1.5 × 1012 (n = 3) vg of rAAV2/8-LSP1-mOTC, and 2 weeks later liver and urine were collected for analysis. Wild-type (n = 7) and untreated Spfash (n = 6) mice were included as positive and negative controls. (a) Western analysis of liver lysates (30 µg protein per lane) from control and vector-treated mice. Actin was included as a loading control. Molecular weight markers are shown (kd). (b) OTC activity in liver lysates from control and vector-treated mice. Fold differences compared to wild-type activity are indicated. (c) Urinary orotic acid normalized against creatinine from control and vector-treated mice. Error bars represent mean ± SEM. OTC, ornithine transcarbamylase; Spfash, OTC-deficient; vg, vector genome; wt, wild-type.