Figure 7. Systemic gene transfer does not affect blood glucose and liver function but induces low antibody levels against the transgene.

(A) Experimental design: 3-month-old KO mice received a single injection of SYS or LS vector at a dose of 2.5 × 10¹³ vg/kg. Age-matched wild-type (WT) and untreated Gaa^–/– (KO) mice were used as controls. The samples were collected 1 month after dosing. (B and C) The levels of blood glucose and conventional hepatic serum biomarkers across the groups (SYS, n = 7; LS, n = 4). (D) Hematoxylin/eosin staining of liver sections. Bar = 100 μm. (E) Serum anti-human GAA IgG in SYS- (n = 9) and LS-treated (n = 6) KO mice. Statistical significance was determined by 1-way ANOVA and unpaired 2-tailed Student’s t test. Graphs represent mean ± SD. *P < 0.05.