Figure 3.

Sustained secretion of therapeutic levels of hAAT from the C-AT vector in either SCID or C57BL mice. (A) The mean total serum levels of hAAT observed in groups of either SCID (squares) or C57BL (circles) mice receiving either low dose (5 × 10¹¹ particles) (open symbols) or high dose (1.4 × 10¹³ particles) (filled symbols) single injections of the C-AT vector measured at time points ranging from 1 to 16 wk after injection. For each strain, the high-dose curve is significantly different from the low-dose curve (P = 0.009 for SCID, P = 0.02 for C57BL), but the strains do not differ from each other. (B) Analogous data with the E-AT vector. None of these differences were significant. (C) An immunoblot of sera taken from several of the C-AT vector-treated mice at 11 wk after vector administration. Ten microliters of a 1:100 dilution of serum was electrophoresed by 10% SDS/PAGE, blotted, and incubated with 1:1,500 dilution of goat anti-hAAT-horseradish peroxidase conjugate (Cappel/ICN). Samples from three high-dose SCID (h1–h3), one high-dose C57BL (h3), and three low-dose C57BL (lo1–lo3) were included, along with one negative control (saline-injected = sal) serum to indicate the level of reactivity with endogenous mAAT. As a standard, hAAT was added either to negative-control C57BL serum (first hAAT lane) or to PBS (second hAAT) lane to a final equivalent serum concentration of 100 μg/ml.