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