Table 3.

Higher AAV neutralizing capacity detected in vivo than in vitro




Percent of 5 × 1011 vg of AAV-hFIX neutralized in vivo
Neutralizing antibody titer
Neutralizing capacity in vitro,*vg
Predicted
Actual§
1:3.3   4.5 × 1010  4.5   100  
1:10
 
9 × 1010
 
9
 
100
 



Percent of 5 × 1011 vg of AAV-hFIX neutralized in vivo
Neutralizing antibody titer
Neutralizing capacity in vitro,*vg
Predicted
Actual§
1:3.3   4.5 × 1010  4.5   100  
1:10
 
9 × 1010
 
9
 
100
 
*

Determined by in vitro neutralizing capacity assay as described in Materials and methods. The neutralizing capacity of a specified titer was defined as the maximal vector dose neutralized by 50% or less

Determined in SCID mice reconstituted with IVIG and intravenously administered vector

The predicted percentage was derived by dividing 50% of in vitro neutralizing capacity (vg) by the injected vector dose of 5 × 1011 vg in vivo

§

As observed in vivo

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