Fig. 2

AAV2/8-hGLA copy numbers and the copy number of GLA mRNA in the livers of AAV2/8-hGLA–treated FD mice. FD mice were intravenously administered formulation buffer or the AAV2/8-hGLA at doses of 0.75E + 12, 1E + 12, 2.5E + 12, and 5E + 12 vg/kg, and tissues were collected at 12 weeks post-injection. A AAV vector genome (vg) copy numbers and B the copy number of GLA mRNA were determined in the liver. Data are presented as the mean ± SEM (n = 4, 5, 5, 6, and 4 for the FD, 0.75E + 12, 1E + 12, 2.5E + 12, and 5E + 12 vg/kg groups, respectively). *P < 0.05, **P < 0.01, ***P < 0.001, ****P < 0.0001. Unpaired t-test was used to compare two groups of data. gDNA = genomic DNA