HCC incidence in AAV-injected and control mice
| Treatment group . | AAV2-hFIX16 dose . | HCC rate, n/N (%) . |
|---|---|---|
| Uninjected | N/A | 1/12 (8.3) |
| Saline | N/A | 0/19 (0) |
| Empty capsid | 1 × 1014 vc/kg | 0/18 (0) |
| Combined controls | 1/49 (2.0) | |
| AAV dose A | 5 × 1012 vg/kg | 2/53 (3.8) |
| AAV dose B | 1 × 1014 vg/kg | 2/16 (12.5) |
| Combined AAV* | 4/69 (5.8) |
| Treatment group . | AAV2-hFIX16 dose . | HCC rate, n/N (%) . |
|---|---|---|
| Uninjected | N/A | 1/12 (8.3) |
| Saline | N/A | 0/19 (0) |
| Empty capsid | 1 × 1014 vc/kg | 0/18 (0) |
| Combined controls | 1/49 (2.0) | |
| AAV dose A | 5 × 1012 vg/kg | 2/53 (3.8) |
| AAV dose B | 1 × 1014 vg/kg | 2/16 (12.5) |
| Combined AAV* | 4/69 (5.8) |
All mice were of the C57BL/6J strain, were 2-3 months old at the time of injection, and were followed for 18 months. The injection route was through the portal vein for those mice receiving injections. Bold indicates combined values.
vc indicates vector capsids; vg, vector genomes; and N/A, not applicable.
P value from 1-tailed Fisher exact test compared HCC rates of combined controls and combined AAV (P = .31).