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 |