Figure 2.

Modeling the phenomenon in vivo

(A) The mechanistic relationship between the AAV dose and the ultimate transgene plasma concentration is highly complex and depends on overall transduction and the physiology of the animal. (B) Derivation of relationship between the steady-state concentration and secretion rate from the overall mass balance. (C) Dose response of steady-state rate in vivo. Black triangles represent mean ± SD for n = 3 mice. Black line: Hill equation dose-response curve fit (Equation 6). Red dotted line: 95% confidence interval. (D) Hill equation and extracted model parameters.