TABLE 2.
Biological activity of nuclear-localized AAV VG
| Vector (purification method) | Day | Nuclear-localized vector copies (VG/DGE)a | hFIX (ng/ml)a | VG activityb |
|---|---|---|---|---|
| AAV2/2 (HPLC) | 1 | 4.03 ± 0.41 | 5.52 ± 0.2 | <2 |
| 3 | 3.90 ± 0.20 | 180 ± 13 | 46 | |
| 7 | 4.18 ± 0.53 | 694 ± 65 | 166 | |
| 21 | 3.78 ± 0.17 | 3,308 ± 302 | 875 | |
| 42 | 2.08 ± 0.09 | 2,188 ± 65 | 1,052 | |
| AAV2/2 (CsCl) | 1 | 4.61 ± 0.41 | 0 | 0 |
| 3 | 5.98 ± 0.20 | 116 ± 18 | 20 | |
| 7 | 4.19 ± 0.53 | 485 ± 32 | 116 | |
| 21 | 2.99 ± 0.17 | 1,451 ± 139 | 485 | |
| 42 | 2.58 ± 0.09 | 3,237 ± 119 | 1,255 | |
| AAV2/6 (CsCl) | 1 | 0.96 ± 0.05 | 6.5 ± 1 | 7 |
| 3 | 0.91 ± 0.08 | 935 ± 140 | 1,027 | |
| 7 | 0.84 ± 0.11 | 2,329 ± 358 | 2,773 | |
| 21 | 1.48 ± 0.17 | 5,627 ± 449 | 3,802 | |
| 42 | 1.53 ± 0.11 | 4,328 ± 275 | 2,828 | |
| AAV2/8 (CsCl) | 1 | 2.99 ± 0.59 | 594 ± 90 | 198 |
| 3 | 4.63 ± 0.677 | 9,739 ± 448 | 2,103 | |
| 7 | 6.90 ± 1.14 | 13,557 ± 2,314 | 1,964 | |
| 21 | 10.24 ± 2.05 | 25,738 ± 1,632 | 2,513 | |
| 42 | 8.56 ± 0.90 | 25,816 ± 519 | 3,015 |
a
Values are means from four animals ± the standard error.
b
Mean hFIX (in nanograms per milliliter) per mean nuclear-localized VG copy number (VG per DGE).