Table 1.
Summary of results in hemophilia B dogs after skeletal muscle delivery of AAV-canine FIX R338L
| Dog | Age/sex/weight | Plateau cFIX expression |
Ratio activity/antigen* | WBCT,† min | Inhibitors | IFN-γ ELISpot |
|||
|---|---|---|---|---|---|---|---|---|---|
| Activity, % | Antigen, % | AAV | cFIX | 338 peptides | |||||
| M55 | 7 mo/male/23 kg | 8 | 1.5 | 8.58 ± 0.46 | 11.4 ± 0.7 | ND | — | — | — |
| M59 | 7 mo/male/16.5 kg | 3.5 | 0.35 | 9.24 ± 0.44 | 13.7 ± 0.7 | ND | ND | ND | ND |
| N07 | 6 mo/male/13 kg | 5.5 | 0.65 | 9.0 ± 0.37 | 14.4 ± 0.7 | ND | ND | ND | ND |
ND indicates not detected; and —, not done.
*
Average ratio of activity/antigen ± SE from all time points.
†
Whole blood clotting time: average values ± SE (normal values, 8-12 minutes).