Summary of canine F.IX antigen and clotting activity in hemophilia B dogs following delivery of AAV2-CMV-cF.IX by isolated limb perfusion or by intravenous injection
. | Age at time of injection, mo . | Gender . | Weight, kg . | Route . | Vector genome/kg . | Circulating cF.IX, ng/mL . | aPTT,*sec . | F.IX*activity, % . | Inhibitor to cF.IX . | Follow-up, mo . |
---|---|---|---|---|---|---|---|---|---|---|
Experimental | ||||||||||
D99 | 15 | Female | 16.7 | ILP | 3.7 × 1012 | 730 ± 60 | 20 ± 2 | 15 ± 2 | No | 39 |
F57 | 4.5 | Male | 12 | ILP | 1.7 × 1012 | 260 ± 52 | 28 ± 4 | 5.5 ± 1.3 | No | 27 |
H08 | 6 | Male | 17.2 | ILP | 3.0 × 1012 | 210 ± 16 | 32 ± 3 | 4.2 ± 1.5 | No | 8 |
Control | ||||||||||
E60† | 6 | Female | 22.5 | ILP | 3.9 × 1012 | 0 | More than 50 | Less than 1 | Yes | ≈ 10 |
E59 | 3 | Female | 14.5 | IV | 2.9 × 1012 | 31-78 | 41 ± 3 | 1 ± 0.4 | No | 37 |
. | Age at time of injection, mo . | Gender . | Weight, kg . | Route . | Vector genome/kg . | Circulating cF.IX, ng/mL . | aPTT,*sec . | F.IX*activity, % . | Inhibitor to cF.IX . | Follow-up, mo . |
---|---|---|---|---|---|---|---|---|---|---|
Experimental | ||||||||||
D99 | 15 | Female | 16.7 | ILP | 3.7 × 1012 | 730 ± 60 | 20 ± 2 | 15 ± 2 | No | 39 |
F57 | 4.5 | Male | 12 | ILP | 1.7 × 1012 | 260 ± 52 | 28 ± 4 | 5.5 ± 1.3 | No | 27 |
H08 | 6 | Male | 17.2 | ILP | 3.0 × 1012 | 210 ± 16 | 32 ± 3 | 4.2 ± 1.5 | No | 8 |
Control | ||||||||||
E60† | 6 | Female | 22.5 | ILP | 3.9 × 1012 | 0 | More than 50 | Less than 1 | Yes | ≈ 10 |
E59 | 3 | Female | 14.5 | IV | 2.9 × 1012 | 31-78 | 41 ± 3 | 1 ± 0.4 | No | 37 |
Activated partial thromboplastin time (aPTT) for normal dogs: 16.5 ± 3.0 seconds; for untreated hemophilia B dogs: longer than 50 seconds.
ILP indicates isolated limb perfusion; IV, intravenous.
Values represent data from several time points (D99 and F57 from 2 to 26 months, H08 from 2 to 8 months)
Animal did not receive cyclophosphamide