Figure 1.
In vivo dose response of a secreted reporter protein at steady-state concentrations
(A) Example of plasma concentration (Cp ng/mL) of GLuc over time following an intravenous AAV injection. Data points represent mean ± SD for n = 4 mice. Solid lines show the fit increasing exponential decay model (Equation 4). Cpss is the steady-state plasma concentration found using the model fit. (B) Plasma concentrations of GLuc at steady state for the indicated AAV dose. Bars represent mean ± SD for n = 3 mice for each dose. (C) Viral genome quantity as measured by qPCR for various tissues from animals injected with 1011.5 vg. Bars represent mean ± SD for n = 3 mice. n.s., not significant; ∗∗∗∗p < 0.0001.