Table 2

Typical inclusion criteria in AAV gene therapy trials, current and proposed

Severe hemophilia B: FIX ≤ 1% 
Age ≥ 18 y 
HCV RNA viral load-negative* 
HIV-negative† 
No previous history of FIX inhibitor 
At least 20 exposure days to FIX concentrate 
Anti–AAV-neutralizing antibody titer ≤ 1:5 
Severe hemophilia B: FIX ≤ 1% 
Age ≥ 18 y 
HCV RNA viral load-negative* 
HIV-negative† 
No previous history of FIX inhibitor 
At least 20 exposure days to FIX concentrate 
Anti–AAV-neutralizing antibody titer ≤ 1:5 
*

Earlier trial included subjects who were HCV RNA viral load-positive, but they are now excluded because of potential need for course of steroids.

†

Some trials may include HIV-positive persons who are stable with adequate CD4 counts on highly active antiretroviral therapy.

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