TABLE 4.
Details of experiment 4a
| Group no. | Pretreatment | No. of mice per group | PHH donor and no. of PHHs per transplant | huAlb (mg/mL) | HBV status | AAV vector and dose (vg/mouse) |
|---|---|---|---|---|---|---|
| 1 | None | 1 | None | 0 | None | scAAV3B-smCBA-GFP, 1012 |
| 2 | None | 1 | None | 0 | None | scAAV.LK03-smCBA-GFP, 1012 |
| 3 | MCT | 3 | A and B, 5 × 105 | >0.1b | 3, infected | scAAV3B-smCBA-GFP, 2 × 1010 |
| 4 | MCT | 3 | A and B, 5 × 105 | >0.1b | 3, infected | scAAV3B-smCBA-GFP, 2 × 1011 |
| 5 | MCT | 3 | A and B, 5 × 105 | >0.1b | 3, infected | scAAV3B-smCBA-GFP, 1012 |
| 6 | MCT | 3 | A and B, 5 × 105 | >0.1b | 3, infected | scAAV.LK03-smCBA-GFP, 2 × 1010 |
| 7 | MCT | 3 | A and B, 5 × 105 | >0.1b | 3, infected | scAAV.LK03-smCBA-GFP, 2 × 1011 |
| 8 | MCT | 3 | A and B, 5 × 105 | >0.1b | 3, infected | scAAV.LK03-smCBA-GFP, 1012 |
a
In this experiment, a single naive (nonhumanized) NSG-PiZ mouse was transduced with the high dose of each AAV vector (groups 1 and 2). A total of 18 male HBV+ mice were randomly assigned to groups 3 to 8 (at least one donor A PHH and one donor B PHH mouse per/group). At day 142 post-HBV infection, mice were administered AAV vectors intravenously at the indicated dose, and at 4 weeks post-AAV administration, livers were analyzed for levels of gene transfer (GFP expression).
b
These huAlb values are from 12 weeks posttransplant. huAlb levels at the time of AAV administration were unknown.