Table 1.

Expression of Human F.IX in CD-1 Mice After Intravenous Injection of AAV-CMV-F.IX at Day 1 of Life

Days Postinjection Expression of hF.IX in Plasma (ng/mL)-150% Hepatocytes Expressing hF.IX-151
77 (±24)  3 (±0.6)  
14 15 (±6)  1.3 (±0.5)  
35  2 (±3.5) ND  
60  0  ND  
240-300  0.25 (±0.1) 
Days Postinjection Expression of hF.IX in Plasma (ng/mL)-150% Hepatocytes Expressing hF.IX-151
77 (±24)  3 (±0.6)  
14 15 (±6)  1.3 (±0.5)  
35  2 (±3.5) ND  
60  0  ND  
240-300  0.25 (±0.1) 

Animals were killed at day 5 (n = 3), day 14 (n = 2), and day 240-300 (n = 8).

Abbreviation: ND, not determined.

F0-150

As determined by ELISA.

F0-151

As determined by image analysis of immunofluorescent staining of tissue sections.