Figure 6.

rAAV Produced Using the Human Manufacturing Platform Has Significantly More Functional Human Liver Transduction In Vivo

(A) Schematic illustrating the production of humanized liver mice used for assessing comparative functional human liver transduction in vivo with rAAV expressing Firefly luciferase (FLuc). (B) In vivo time course functional transduction assays comparing human and baculovirus (baculo-)Sf9-produced ssAAV1-EF1α-FLuc after i.v. tail vein administration (5e10 vg/mouse) in age-matched humanized liver mice. Mean radiance (photons/s/cm²/sr) is displayed with all mice imaged on their ventral side on the same shared scale. (C) Quantification of rAAV1 FLuc radiance in male human liver. Each symbol indicates mean signal (±SD) from four humanized mice. (D) Quantification of rAAV1 FLuc radiance in female human liver. Each symbol indicates mean signal (±SD) from three humanized mice. ∗p ≤ 0.05, ∗∗p ≤ 0.01, ∗∗∗p ≤ 0.001, ∗∗∗∗p ≤ 0.0001.