Fig. 4: AAV-GAA gene transfer results in higher central nervous system uptake of GAA and glycogen clearance compared to enzyme replacement therapy (ERT). | Nature Communications

Fig. 4: AAV-GAA gene transfer results in higher central nervous system uptake of GAA and glycogen clearance compared to enzyme replacement therapy (ERT).

From: Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates

Fig. 4

A GAA activity in brain and B spinal cord at sacrifice. C–F Analysis of lysosomal GAA enzyme at sacrifice in brain (C, D) and spinal cord (E, F) of mice treated with ERT or AAV-GAA gene transfer at 1 × 1011 vg/kg. D, F Quantification of the lysosomal GAA bands in C and E. G, H Analysis of glycogen content in brain (G) and spinal cord (H). Data shown as average ± SD. Statistical analysis: A, B, G, H One-way ANOVA with Tukey post-hoc test; D, F t-test. *, # and # p < 0.05; ** and †† p < 0.01; *** and ### p < 0.001; ****, †††† and #### p < 0.0001. Exact p values for A, B, G, H are provided in the Source Data file. A, B, D, F–H: ERT, n = 9; AAV-GAA, n = 7 per AAV dose; Gaa+/+Cd4−/− PBS, n = 7, and Gaa−/−Cd4−/− PBS, n = 7.

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