Table 2.

F.IX-specific activity as a function of expression levels at several time points following intramuscular injection of AAV vectors



Specific activity for F.IX range, mean ± SEM
AAV-F.IX
50-499 U/mg F.IX, ng/mL
500-1500 U/mg F.IX, ng/mL
WT   123 ± 15   15 ± 2  
K5A/V10K   112 ± 16   49 ± 11* 
R338A
 
305 ± 48
 
24 ± 11
 


Specific activity for F.IX range, mean ± SEM
AAV-F.IX
50-499 U/mg F.IX, ng/mL
500-1500 U/mg F.IX, ng/mL
WT   123 ± 15   15 ± 2  
K5A/V10K   112 ± 16   49 ± 11* 
R338A
 
305 ± 48
 
24 ± 11
 

ANOVA was used for statistical analysis Tukey-Kramer comparison test between all groups. Data are mean values ± SEM for specific activities.

*

K5A/V10K compared with WT (P < .01)

R338A compared with WT or K5A/V10K (P < .0001)

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