Figure 1.

Comparison of Liver Depot Gene Therapy with ERT

In a 5-week study, GAA-KO mice were treated with (A) either a weekly injection of rhGAA (ERT; 20 mg/kg; n = 10) or a single injection of AAV2/8-LSPhGAApA (AAV; 8 × 10¹¹ vg/kg; n = 10). (B–F) The endpoints included: (B) GAA activity and (C) glycogen content in the tissues, (D) antibody formation, (E) blood GAA, and (F) heart size. GAA activity was increased and glycogen content was reduced in the heart and skeletal muscles. Mean ± SD is shown. *p < 0.05, **p < 0.01, ***p < 0.001, ****p < 0.0001 from ANOVA.