Table 3.

Quantification of plasma enzymes to assess hepatotoxicity




No. subjects

Albumin, g/L

ALT, U/L

AST, U/L

GGT, U/L

ALP, U/L

Bile acids, μM
hFIX lentivirus   6   32.6 ± 0.6   95.5 ± 15.9   125.8 ± 9.1   7.2 ± 0.7   129.8 ± 8.4   47.0 ± 5.2  
Untreated   5   32.6 ± 0.8   84.4 ± 23.5   109.8 ± 24.6   7.8 ± 1.2   146.6 ± 6.0   42.3 ± 5.1  
hFIX adenovirus
 
2
 
33.8, 34
 
111, 113
 
148, 188
 
8, 9
 
306, 259
 
53.5, 48
 



No. subjects

Albumin, g/L

ALT, U/L

AST, U/L

GGT, U/L

ALP, U/L

Bile acids, μM
hFIX lentivirus   6   32.6 ± 0.6   95.5 ± 15.9   125.8 ± 9.1   7.2 ± 0.7   129.8 ± 8.4   47.0 ± 5.2  
Untreated   5   32.6 ± 0.8   84.4 ± 23.5   109.8 ± 24.6   7.8 ± 1.2   146.6 ± 6.0   42.3 ± 5.1  
hFIX adenovirus
 
2
 
33.8, 34
 
111, 113
 
148, 188
 
8, 9
 
306, 259
 
53.5, 48
 

Serum was taken 204 to 331 days after fetal intravascular injection of hFIX lentivirus into MF1 mice (indicated by the open squares in Figure 1C). Untreated mice were used as negative controls, and blood collected 24 hours after adult injection of hFIX-adenovirus was used as a positive control. Lentivirus-treated mice showed no evidence of liver toxicity, unlike the adenovirus-treated mice. Values are mean ± SEM, except in the third row, where data are raw values from 2 mice.

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