Fig. 5: α-Gal A enzyme activity levels in the liver, kidney and heart of Fabry mice administered AAV8-FLT190 2 × 1012 vg/kg. | Gene Therapy

Fig. 5: α-Gal A enzyme activity levels in the liver, kidney and heart of Fabry mice administered AAV8-FLT190 2 × 1012 vg/kg.

From: Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease

Fig. 5

α-Gal A enzyme activity was measured using the fluorogenic substrate 4-methylumbelliferyl-α-D-galactopyranoside. Data are mean ± SD, n = 4 to 6 animals per time point. One-way ANOVA with Bonferroni correction was used to compare mean of all treatment groups (AAV8-FLT190 treated vs. untreated (vehicle) controls, p < 0.0001). Fold increase was calculated based on WT untreated controls (n = 4). α-Gal A = alpha galactosidase A; AAV8 = adeno-associated virus serotype 8; SD = standard deviation; vg = vector genomes; WT = wild type.

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