Gene transfer after liver-targeted delivery of rAAV CAGG-FIX in rhesus macaque monkeys
| Monkey . | Weight, kg . | Delivery route . | Infusion* . | Transgene copy no./diploid genome . | Maximum hFIX expression (% of normal) . | Anti-hFIX Abs . | Duration of expression, wks . |
|---|---|---|---|---|---|---|---|
| 1 | 4.6 | HA | 1 | 0.05 | 0.8 | No | < 15 |
| 2 | 4.6 | HA | 1 | 0.1 | 1.0 | No | < 15 |
| 3 | 2.2 | HA | 2 | 3.2 | 4 | No | > 50 |
| 4 | 3.8 | PV | 1 | 40 | 10 | No | > 40 |
| 5 | 7.6 | PV | 2 | 25 | 3 | Yes | < 3 |
| Monkey . | Weight, kg . | Delivery route . | Infusion* . | Transgene copy no./diploid genome . | Maximum hFIX expression (% of normal) . | Anti-hFIX Abs . | Duration of expression, wks . |
|---|---|---|---|---|---|---|---|
| 1 | 4.6 | HA | 1 | 0.05 | 0.8 | No | < 15 |
| 2 | 4.6 | HA | 1 | 0.1 | 1.0 | No | < 15 |
| 3 | 2.2 | HA | 2 | 3.2 | 4 | No | > 50 |
| 4 | 3.8 | PV | 1 | 40 | 10 | No | > 40 |
| 5 | 7.6 | PV | 2 | 25 | 3 | Yes | < 3 |
HA indicates hepatic artery; and PV, portal vein.
Vector particles (4 × 1012 vgs/kg) were given either as 1 infusion or in 2 equal divided doses separated by 3 days.