Table 4.

Summary of hemophilia B dogs injected intramuscularly with AAV-1 or AAV-2 vector

Dog
Age, mos.
Sex
AAV serotype
Total vector genome
Vector genome per kg
Vector dose per site
Maximum cF.IX level, ng/mL
Anti-cF.IX, Bethesda units
E35   10   female   AAV-1   1.6 × 1013  1.0 × 1012  5.0 × 1011  87*  8-15 BU  
E57†  3   male   AAV-1   2.0 × 1012  2.4 × 1011  6.8 × 1010  104   2-8.5 BU  
B45   2   male   AAV-2   7.4 × 1011  1.3 × 1011  4.1 × 1010  2.6   none  
B48   8   female   AAV-2   6.8 × 1013  3.4 × 1012  1.1 × 1012  20   none  
D32†  2.5   male   AAV-2   2.5 × 1013  5.6 × 1012  2.0 × 1012  40   none  
D31   1.5   male   AAV-2   3.2 × 1013  8.5 × 1012  2.0 × 1012  39   none  
B85   3   female   AAV-2   5.0 × 1013  8.5 × 1012  2.1 × 1012  70   6.8 BU, t-ransient  
B14
 		 23
 		 male
 		 AAV-2
 		 2.3 × 1014
 		 1.1 × 1013
 		 1.2 × 1013
 		 30*
 		 ≤ 24.5 BU
 
Dog
Age, mos.
Sex
AAV serotype
Total vector genome
Vector genome per kg
Vector dose per site
Maximum cF.IX level, ng/mL
Anti-cF.IX, Bethesda units
E35   10   female   AAV-1   1.6 × 1013  1.0 × 1012  5.0 × 1011  87*  8-15 BU  
E57†  3   male   AAV-1   2.0 × 1012  2.4 × 1011  6.8 × 1010  104   2-8.5 BU  
B45   2   male   AAV-2   7.4 × 1011  1.3 × 1011  4.1 × 1010  2.6   none  
B48   8   female   AAV-2   6.8 × 1013  3.4 × 1012  1.1 × 1012  20   none  
D32†  2.5   male   AAV-2   2.5 × 1013  5.6 × 1012  2.0 × 1012  40   none  
D31   1.5   male   AAV-2   3.2 × 1013  8.5 × 1012  2.0 × 1012  39   none  
B85   3   female   AAV-2   5.0 × 1013  8.5 × 1012  2.1 × 1012  70   6.8 BU, t-ransient  
B14
 		 23
 		 male
 		 AAV-2
 		 2.3 × 1014
 		 1.1 × 1013
 		 1.2 × 1013
 		 30*
 		 ≤ 24.5 BU
 

AAV-2-treated dogs previously reported (see Herzog et al10  [1999] and Herzog et al11  [2002]).

*

Maximum cF.IX levels detected before antibody formation to F.IX.

†

Dogs treated with cyclophosphamide, 1 dose per week for 6 weeks.

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