Fig. 2.

Significant but sub-therapeutic expression of human α₁-antitrypsin following non-viral gene transfer to the lung. BALB/c mice were treated with one or six doses of GL67:phCEFI-sohAAT (80 μg/dose/mouse) or negative control (PBS) by nasal sniffing. Animals were harvested 6 days after the final dose and human α₁-antitrypsin (hAAT) expression quantified in a lung tissue homogenate and b epithelial lining fluid (ELF). Each data point represents one animal; horizontal bars represent group medias. ** = p < 0.01. ns = not significant (Kruskal–Wallis test followed by Dunn multiple comparison post hoc test). c In a separate experiment BALB/c mice were treated with GL67A complexed with plasmids carrying Firefly luciferase (Lux; pCIK-Lux) or sohAAT (pCIK-sohAAT) cDNAs. Lungs were harvested 48 h post transduction, and qRT-PCR performed on total RNA. Data are expressed as percentage vector mRNA compared with endogenous mRNA (murine cystic fibrosis transmembrane conductance regulator, mCftr). Each data point represents one animal. Horizontal bars represent group medians. PBNQ = positive but not quantifiable, ns = not significant