The Pharmacological Chaperone AT2220 Increases Recombinant Human Acid α-Glucosidase Uptake and Glycogen Reduction in a Mouse Model of Pompe Disease
Figure 3
AT2220 increases the circulating levels of rhGAA in GAA KO mice.
Twelve-week old male GAA KO mice were administered vehicle (water) or AT2220 (30 mg/kg) via oral gavage once every other week for 8 weeks. Thirty minutes after each AT2220 oral administration, vehicle (saline) or rhGAA (20 mg/kg) was administered via bolus tail vein injection. Blood was collected after the last (i.e., 4th) rhGAA administration and, GAA activity (upper panel) and protein levels (lower panel) were measured in plasma as described in ‘Materials and Methods’. Each bar represents the mean±SEM of the GAA activity measured from 5 mice per group. Statistically significant increases were seen in plasma GAA activity compared to baseline (*p<0.05, t-test) and compared to rhGAA administration alone (#p<0.05, t-test). Each lane on the Western blot contains plasma from a single mouse, and is representative of two mice in each group.