Effect of AT2220 incubation on enzyme activity and protein levels of secreted wild-type and mutant GAA.

COS-7 cells were transfected with wild-type or various mutant forms of GAA, followed by incubation with or without 100 µM AT2220. Increases in the enzymatic activity and total protein levels of secreted GAA were measured as described in ‘Materials and Methods’, and as shown in Fig. 2. AT2220-mediated increases in the relative specific activities of the various mutant forms of GAA were calculated as the ratio of GAA enzyme activity to the total GAA protein levels. Activity and protein values represent the mean ± SD from four independent experiments.