Fig. 1.

AAV2/8-hGLA produces continuous supraphysiological levels of glycosylation-modified α-Gal A in the plasma of FD mice. A α-Gal A activity was determined in the plasma of FD mice treated with different doses of AAV2/8-hGLA. Data are presented as the mean ± SEM (n = 2–12). Plasma was collected through tail vein bleeding at the indicated times, and the enzyme activity was measured using an α-Galactosidase (α-GAL) Activity Assay Kit. The dotted line indicates plasma α-Gal A at steady state levels in the untreated FD mice. B α-Gal A protein glycosylation patterns of plasma were determined in the plasma of 2.5E + 12 vg/kg AAV2/8-hGLA–treated FD mice; representative Western blot analyses are shown. Rec.GLA (an α-Gal A expressed in CHO mammalian cells) was used as a positive control for glycosylation analysis. PNGase F = peptide:N-glycosidase F; Endo H = endoglycosidase H