Summary of F.IX levels and coagulation parameters in C57B1/6 hemophilia B mice 4 weeks following AAV-F.IX vector delivery to liver by tail-vein injection at doses of 4 × 1012 vg/kg
AAV-F.IX . | No. mice . | F.IX Ag, ng/mL . | F.IX clotting activity, % . | TAT levels, ng/mL . | Blood loss, OD . |
|---|---|---|---|---|---|
| WT | 4 | 491 ± 135 | 9.5 ± 2.3 | 14.3 ± 2.8 | 0.46 ± 0.04 |
| R338A | 6 | 451 ± 63 | 47.4 ± 14.2* | 29.3 ± 11.3 | 0.22 ± 0.04* |
AAV-F.IX . | No. mice . | F.IX Ag, ng/mL . | F.IX clotting activity, % . | TAT levels, ng/mL . | Blood loss, OD . |
|---|---|---|---|---|---|
| WT | 4 | 491 ± 135 | 9.5 ± 2.3 | 14.3 ± 2.8 | 0.46 ± 0.04 |
| R338A | 6 | 451 ± 63 | 47.4 ± 14.2* | 29.3 ± 11.3 | 0.22 ± 0.04* |
TAT levels in hemophilia B mice (n = 10) prior vector injection was 4 ± 2 ng/mL. Hemostatically normal C57B1/6 mice (n = 15) presented TAT levels of 29.4 ± 8.5 ng/mL.
Blood loss (mean ± SD) during the tail-clipping assay was measured by the hemoglobin absorbance A575 in the saline in which tails were immersed.
Blood loss for normal C57/B16 mice was 0.07 ± 0.02, whereas for untreated hemophilia B mice values were 1.0 ± 0.36.
Student ttest was used for comparison between F.IX-WT and R338A; P < .005. OD indicates optical density