Table 1.

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

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