Figure 7.

Functional Asparagine Substitutions at Non-NG Sites with High Variability between Lots

(A) Titers of WT AAV8 and mutant vectors were produced by small-scale triple transfection in 293 cells, as measured by qPCR. Transduction efficiencies for crude vector material were measured as described in Figure 4. Titers and transduction efficiencies were normalized to the value of the WT AAV8 control. (B) Representative luciferase images at day 14 post-injection are shown for mice receiving WT AAV8.CB7.ffluc and N499Q capsid mutant vector. (C) Luciferase expression on day 14 of the study period from C57BL/6 mice injected intravenously with WT AAV8 or mutant vectors (n = 3 or 4) was measured by luciferase imaging and reported in total flux units. WT control shared with Figure 6C. All data are represented as mean + SD.