Table 1.
Rhesus Limb Infusion Experimental Design
| Dose Group | Animal # (Sex; NAb titer) | Test Article (6 × 1012 vg/kg for All Groups) | Route | Dosing Volume |
|---|---|---|---|---|
| IM-AAV1 | RA1683 (F; 1:5) | rAAV1-CB-rhAATmyc | IM | 0.5 mL per injection (8 injections) |
| RA1598 (M; 1:10) | ||||
| IAPD-AAV1 | RA1709 (F; 1:5) | rAAV1-CB-rhAATmyc | IAPD | 12.5 mL/kg |
| RA1562 (M; 1:10) | ||||
| IAPD-AAV8 | RA1660 (F; 1:10) | rAAV8-CB-rhAATmyc | IAPD | 12.5 mL/kg |
| RA1664 (F; 1:10) | ||||
| VLP-AAV1 | RA0770 (F; <1:5) | rAAV1-CB-rhAATmyc | VLP | 50 mL/kg |
| RA1567 (M; 1:10) | ||||
| VLP-AAV8 | RA1676 (F; 1:10) | rAAV8-CB-rhAATmyc | VLP | 50 mL/kg |
| RA1703 (F; 1:10) |
All dosing was performed in the right hindlimb. Intramuscular dosing was performed as a total of eight injections, four into the right quadriceps and four into the right gastrocnemius. NAb, neutralizing antibody titer (anti-AAV1 or anti-AAV8 titer listed depending on the vector delivered to that animal); IM, intramuscular; F, female; M, male; CB, chicken beta-actin promoter with a CMV enhancer; rhAATmyc, rhesus alpha-1 antitrypsin gene with a c-myc tag; IAPD, intra-arterial push and dwell; VLP, venous limb perfusion;