Fig. 5: rAAV8 transduction of liver and spleen of Lipa−/− mice results in long-term increase of LAL activity and correction of hepatosplenomegaly.
From: Rescue of lysosomal acid lipase deficiency in mice by rAAV8 liver gene transfer

rAAV8 transduction level in (a) liver and (b) spleen at indicated time points (weeks) (n = 4-10). vg vector genome. Fold change of LAL activity over WT mice in (c) liver and (d) spleen at indicated time points (weeks). Weight of (e) liver and (f) spleen at indicated time points (weeks). g Representative images of liver and spleen at 12-weeks post-injection. Bars indicate mean ± SD (n = 4-10) and statistical significance was calculated using two-way ANOVA with Tukey’s test (*p < 0.033; **p < 0.002; ***p < 0.0002). Exact p value is indicated in Supplementary Data 1.