Fig. 3: α-Gal A activity in plasma following administration of AAV8-FLT190 or ERT in wild-type (WT) mice.
From: Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease
A Time course of α-Gal A enzyme activity levels in the plasma of WT mice that received AAV8-FLT190 6 x 1011, 2 × 1012 or 6 x 1012 vg/kg. Data are mean ± SD, n = 4–8 animals per time point. Plasma was collected by tail vein bleeding at the indicated times and the enzyme activity was measured using the fluorogenic substrate 4-methylumbelliferyl-α-D-galactopyranoside. B Plasma clearance curves after intravenous injection of agalsidase alfa at 0.2 mg/kg, in male WT mice. ERT pharmacokinetics using one-phase decay model data are mean ± SD, n = 5 animals per time point. Dotted line indicates plasma α-Gal A at steady state levels following AAV8-FLT190 in wild-type mice. α-Gal A = alpha galactosidase A; AAV8 = adeno-associated virus serotype 8; ERT = enzyme replacement therapy; SD = standard deviation; vg = vector genomes; WT = wild type.
