Fig. 4: rAAV8 administration increases survival and body weight of male Lipa−/− mice and corrects haematological parameters and dyslipidaemia for 34-weeks post-injection.
From: Rescue of lysosomal acid lipase deficiency in mice by rAAV8 liver gene transfer
a Percentage of mice survival. b Mice weight (gr: grams) was recorded every week (n = 6-11). c Linear slopes of mice weight. Bars indicate mean ± SD (n = 3-4) and statistical significance was calculated using one-way ANOVA with Turkey’s Test (*p < 0.033; **p < 0.002; ***p < 0.0002). Exact p value is indicated in Supplementary Data 1. d Fold change of LAL activity over WT mice measured in plasma samples every 2-weeks post-injection (n = 3-11). e Blood cell populations in blood samples collected at 30-weeks post-injection (n = 3-6). f White blood cell (WBC) and (g) platelet counts in blood samples collected every 2-weeks post-injection (n = 1-20). Quantification of (h) ALT (U/L, unit per liter) and (i) HDL in plasma samples collected every 2-weeks post-injection (n = 1-20). Bars indicate mean ± SD 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.
