Figure 2.

AAV2/8-LSPhGAApA Reduced the Glycogen Content of Muscle and Increased the Benefit from Simultaneous ERT

(A) GAA-KO mice were treated with the AAV2/8-LSPhGAA vector (AAV), either with or without simultaneous ERT for four doses (rhGAA, 20 mg/kg every 2 weeks). Number of mice per group was as follows: (−)ERT: 0 (n = 8), 2 × 10¹⁰ (n = 8), 8 × 10¹⁰ (n = 9), 2 × 10¹¹ (n = 8); (+)ERT: 0 (n = 10), 2 × 10¹⁰ (n = 9), 8 × 10¹⁰ (n = 9), 2 × 10¹¹ (n = 9). Biochemical correction in GAA-KO mice was evaluated 8 weeks following gene therapy, either with (shaded bars) or without (black bars). GAA activity and glycogen content shown for (B) heart, (C) diaphragm, and (D) quadriceps. Mean ± SD is shown. *p < 0.05, **p < 0.01, ***p < 0.001, ****p < 0.0001 from two-way ANOVA.