hFIX measurements in test and control mice
Mouse . | Days after injection . | hFIX:C, U/mL . | hFIX:Ag, U/mL . | Days after injection . | hFIX:C, U/mL . | hFIX:Ag, U/mL . | Days after injection . | hFIX:C, U/mL . | hFIX:Ag, U/mL . |
|---|---|---|---|---|---|---|---|---|---|
| Lentivirus | |||||||||
| M1 | 45 | 0.49 | 0.12 | 188 | 1.40 | 0.24 | 297 | 1.25 | 0.18 |
| M2 | 45 | 0.68 | 0.096 | 188 | 0.59 | 0.14 | 297 | 0.80 | 0.16 |
| M3 | 52 | 0.33 | 0.071 | 195 | 0.38 | 0.088 | 304 | 0.50 | 0.11 |
| Adenovirus | |||||||||
| A1 | 45 | 0.14 | 0.039 | 188 | 0.07 | <0.003 | ND | ND | ND |
| A2 | 45 | 0.21 | 0.042 | 188 | 0.08 | <0.003 | ND | ND | ND |
| A3 | 45 | 0.15 | 0.029 | 188 | 0.06 | <0.003 | ND | ND | ND |
| A4 | 101 | <0.02 | 0.016 | 244 | 0.07 | 0.004 | ND | ND | ND |
| A5 | 101 | <0.02 | 0.016 | 244 | 0.06 | <0.003 | ND | ND | ND |
| A6 | 101 | <0.02 | .009 | 244 | 0.08 | <0.003 | ND | ND | ND |
| A7* | 101 | <0.02 | 0.009 | ND | ND | ND | ND | ND | ND |
| Uninjected controls | |||||||||
| C1 | — | 0.07 | <0.003 | ND | ND | ND | ND | ND | ND |
| C2 | — | 0.07 | <0.003 | ND | ND | ND | ND | ND | ND |
| C3 | — | 0.08 | <0.003 | ND | ND | ND | ND | ND | ND |
| C4 | — | <0.02 | <0.003 | ND | ND | ND | ND | ND | ND |
| C5 | — | <0.02 | <0.003 | ND | ND | ND | ND | ND | ND |
Mouse . | Days after injection . | hFIX:C, U/mL . | hFIX:Ag, U/mL . | Days after injection . | hFIX:C, U/mL . | hFIX:Ag, U/mL . | Days after injection . | hFIX:C, U/mL . | hFIX:Ag, U/mL . |
|---|---|---|---|---|---|---|---|---|---|
| Lentivirus | |||||||||
| M1 | 45 | 0.49 | 0.12 | 188 | 1.40 | 0.24 | 297 | 1.25 | 0.18 |
| M2 | 45 | 0.68 | 0.096 | 188 | 0.59 | 0.14 | 297 | 0.80 | 0.16 |
| M3 | 52 | 0.33 | 0.071 | 195 | 0.38 | 0.088 | 304 | 0.50 | 0.11 |
| Adenovirus | |||||||||
| A1 | 45 | 0.14 | 0.039 | 188 | 0.07 | <0.003 | ND | ND | ND |
| A2 | 45 | 0.21 | 0.042 | 188 | 0.08 | <0.003 | ND | ND | ND |
| A3 | 45 | 0.15 | 0.029 | 188 | 0.06 | <0.003 | ND | ND | ND |
| A4 | 101 | <0.02 | 0.016 | 244 | 0.07 | 0.004 | ND | ND | ND |
| A5 | 101 | <0.02 | 0.016 | 244 | 0.06 | <0.003 | ND | ND | ND |
| A6 | 101 | <0.02 | .009 | 244 | 0.08 | <0.003 | ND | ND | ND |
| A7* | 101 | <0.02 | 0.009 | ND | ND | ND | ND | ND | ND |
| Uninjected controls | |||||||||
| C1 | — | 0.07 | <0.003 | ND | ND | ND | ND | ND | ND |
| C2 | — | 0.07 | <0.003 | ND | ND | ND | ND | ND | ND |
| C3 | — | 0.08 | <0.003 | ND | ND | ND | ND | ND | ND |
| C4 | — | <0.02 | <0.003 | ND | ND | ND | ND | ND | ND |
| C5 | — | <0.02 | <0.003 | ND | ND | ND | ND | ND | ND |
Hemophilic fetal mice were injected intravascularly at 15 days of gestation with hFIX-lentivirus or hFIX-adenovirus. At up to 3 time points (shown as open symbols in Figure 1A-B), blood was collected for in vitro FIX:C (coagulation) assay. These values were tabulated alongside the values of hFIX antigen (hFIX:Ag) determined by ELISA (open symbols in Figure 1A-B). Lentivirus-treated mice had high hFIX:C values at all 3 time points, whereas adenovirus-treated mice had only low and transient hFIX:C values. hFIX:C values in untreated hemophilic mice were very low or below the limit of detection.
ND indicates not done.
Mouse A7 died after the first bleed.