Figure 7. In vivo levels of human AAT in ELF over time following administration of the AAV8 vectors.

C57BL/6 mice were administered 8/AMM, 8/AVL, or 8/Null (4 × 10¹¹ gc) by the IV and IPL routes (n = 4–5/group). Lung ELF was collected after sacrifice at 0, 4, 12, or 24 weeks, and human AAT was quantified by ELISA. (A) Male mice, IV. (B) Male mice, IPL. (C) Female mice, IV. (D) Female mice, IPL. At 24 weeks, there was no significant (ns) difference between 8/AMM and 8/AVL for either sex or administration route using Student’s t test. (E and F) Ratio of ELF to serum AAT. (E) Male mice. (F) Female mice. For E and F, data from all doses and time points were combined, and AAT levels (per mg protein) were collectively compared in ELF and serum for mice administered 8/AVL. Statistical analysis was performed using Student’s t test.