Figure 5.

Liver Gene Transfer Eradicates Humoral Immune Response Triggered by Multiple rhGAA Infusions

GAA-KO mice from the three groups were injected with 20 mg/kg rhGAA and 8 × 10¹¹ vg/kg AAV8-LSPhGAA as indicated in the experimental plan (Figure 4A). Scatterplots show total IgG (A), IgG1 (B), and ranked IgG (C) and IgG1 (D), which was measured in plasma 36 weeks after vector injection and ranked by the degree of antibody production. Antibodies were ranked as follows: <0.25 = 0, 0.25–0.5 = 1, 0.51–0.75 = 2, 0.76–1 = 3, and >1 = 4 (Table S1). The quantification of antibody isotypes has been performed using purified mouse IgG isotypes as standard. Mean ± SD is shown. *p < 0.05, **p < 0.01 from ANOVA.