Plasma levels of human FVIII in NOD/SCID mice
| Treatment group . | Plasma level of human FVIII in ng/mL (nM) . | ||||
|---|---|---|---|---|---|
| Day 9 . | Day 28 . | Day 47 . | Day 97 . | Day 156 . | |
| No. cells given | −3 (2) | −4 (2) | −2 (1) | 0 (1) | |
| 20 × 104wtBOEC | 1 (2) | 9 (2) | 11 (2) | 17 (2) | 9 (2) |
| 20 × 104gfpBOEC | 1 ± 4 (3) | 7 ± 5 (3) | 12 ± 7 (3) | 26 ± 4 (3) | 7 ± 4 (3) |
| 5 × 104 tBOEC | 9 (2) | 14 (2) | 45 (2) | 53 (2) | 58 (2) |
| 10 × 104 tBOEC | 9 ± 3 (3) | 19 ± 6 (3) | 57 (1) | 49 (1) | 145 (1) |
| 20 × 104 tBOEC | 16 ± 11 (4) | 39 ± 24 (4) | 114 ± 65 (4) | 146 ± 96 (4)† | 581 ± 517 (3)* |
| 40 × 104 tBOEC | 35 ± 26 (3) | 368 ± 50 (3)* | 534 ± 363 (3)* | 246 (2)‡ | 346 (1) |
| Treatment group . | Plasma level of human FVIII in ng/mL (nM) . | ||||
|---|---|---|---|---|---|
| Day 9 . | Day 28 . | Day 47 . | Day 97 . | Day 156 . | |
| No. cells given | −3 (2) | −4 (2) | −2 (1) | 0 (1) | |
| 20 × 104wtBOEC | 1 (2) | 9 (2) | 11 (2) | 17 (2) | 9 (2) |
| 20 × 104gfpBOEC | 1 ± 4 (3) | 7 ± 5 (3) | 12 ± 7 (3) | 26 ± 4 (3) | 7 ± 4 (3) |
| 5 × 104 tBOEC | 9 (2) | 14 (2) | 45 (2) | 53 (2) | 58 (2) |
| 10 × 104 tBOEC | 9 ± 3 (3) | 19 ± 6 (3) | 57 (1) | 49 (1) | 145 (1) |
| 20 × 104 tBOEC | 16 ± 11 (4) | 39 ± 24 (4) | 114 ± 65 (4) | 146 ± 96 (4)† | 581 ± 517 (3)* |
| 40 × 104 tBOEC | 35 ± 26 (3) | 368 ± 50 (3)* | 534 ± 363 (3)* | 246 (2)‡ | 346 (1) |
Data are expressed as mean (number of mice contributing). SD is shown if n > 2. Cell dose is indicated as the number of cells per injection; 3 injections were administered.
P values are compared to the baseline measurement for the whole group.
BOEC prefixes are as follows: wt indicates unmanipupated BOECs; gfp, BOECs transduced to express GFP only; and t, BOECs stably transfected to express eGFP-FVIII.
P < .001.
P < .05.
P < .01.