Summary of Results in Rats
| Rat No. . | hFX Antigen . | Functional hFX† . | Percentage of hFX That Functions‡ . | Transduction Efficiencyρ . | Normalized Expression . |
|---|---|---|---|---|---|
| . | (μg/mL)* . | . | . | . | (μg hFX/mL/1% transduction)¶ . |
| 113 | 1.1 ± 0.5 (14) | 17.25 ± 0.75 | 123 ± 15 | 0.3 ± 0.1 | 3.67 ± 1.29 |
| 114 | 2.3 ± 0.1 (29) | 54 ± 10.7 | 174 ± 35 | 1.5 ± 0.5 | 1.53 ± 0.52 |
| 115 | 0.88 ± 0.06 (11) | 9.5 ± 0.5 | 79 ± 7 | 1 ± 0.5 | 0.88 ± 0.44 |
| 119 | 2.71 ± 0.1 (34) | 27 ± 3.2 | 108 ± 13 | 1.1 ± 0.5 | 2.46 ± 1.23 |
| Rat No. . | hFX Antigen . | Functional hFX† . | Percentage of hFX That Functions‡ . | Transduction Efficiencyρ . | Normalized Expression . |
|---|---|---|---|---|---|
| . | (μg/mL)* . | . | . | . | (μg hFX/mL/1% transduction)¶ . |
| 113 | 1.1 ± 0.5 (14) | 17.25 ± 0.75 | 123 ± 15 | 0.3 ± 0.1 | 3.67 ± 1.29 |
| 114 | 2.3 ± 0.1 (29) | 54 ± 10.7 | 174 ± 35 | 1.5 ± 0.5 | 1.53 ± 0.52 |
| 115 | 0.88 ± 0.06 (11) | 9.5 ± 0.5 | 79 ± 7 | 1 ± 0.5 | 0.88 ± 0.44 |
| 119 | 2.71 ± 0.1 (34) | 27 ± 3.2 | 108 ± 13 | 1.1 ± 0.5 | 2.46 ± 1.23 |
Plasma was obtained at 6 weeks after transduction. For all columns, the result ± the standard deviation is shown.
The hFX antigenic activity in micrograms per milliliter is shown, with the percentage of normal human levels shown in parentheses.
The hFX functional activity was determined and is reported as the percentage of normal human activity.
The percentage of hFX that functions was determined by dividing the hFX functional activity by the hFX antigen.
ρ Transduction efficiency was determined by using the PCR-based assay shown in Fig 3A. The percentage of liver cells that were transduced was calculated by averaging the results obtained from three separate assays.
¶ Normalized expression was obtained by dividing the hFX antigen levels by the transduction efficiency.