Fig. 2: rAAV8 transduction of liver and spleen of Lipa−/− mice increases LAL activity and corrects hepatosplenomegaly.
From: Rescue of lysosomal acid lipase deficiency in mice by rAAV8 liver gene transfer
rAAV8 transduction level for different vector doses at 12-weeks post injection (a) in liver and (b) spleen. vg vector genome. Fold change of LAL activity over WT mice (c) in liver and (d) spleen for different vector doses. Weight of (e) liver and (f) spleen at 12-weeks post-injection for different vector doses. g Representative images of liver and spleen at 12-weeks post-injection. Ruler indicates centimetres. Bars indicate mean ± SD (n = 3-4) 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.
