Table 3. Comparison of F.IX antigen and... | American Society of Hematology

Table 3.

Comparison of F.IX antigen and biologic activity in C57B1/6 hemophilia B/CD4 knock-out mice 4 weeks following intramuscular injection of AAV vectors encoding F.IX-WT and variants

F.IX	Vector dose	No. mice	F.IX-Ag, ng/mL	F.IX clotting activity, %	TAT levels, ng/mL
R338A	4 × 10¹²	4	213 ± 42	6.3 ± 1.5	76 ± 26^*
WT	4 × 10¹²	4	186 ± 61	3.1 ± 0.3	31 ± 19
K5A/V10K	1 × 10¹²	4	232 ± 29	3.3 ± 0.6	33 ± 25
Hemophilia B/CD4 mice	NA	12	UD	< 1	10 ± 4^*
Hemostatically normal C57B1/6 mice	NA	15	2500†	100	29 ± 9

F.IX	Vector dose	No. mice	F.IX-Ag, ng/mL	F.IX clotting activity, %	TAT levels, ng/mL
R338A	4 × 10¹²	4	213 ± 42	6.3 ± 1.5	76 ± 26^*
WT	4 × 10¹²	4	186 ± 61	3.1 ± 0.3	31 ± 19
K5A/V10K	1 × 10¹²	4	232 ± 29	3.3 ± 0.6	33 ± 25
Hemophilia B/CD4 mice	NA	12	UD	< 1	10 ± 4^*
Hemostatically normal C57B1/6 mice	NA	15	2500†	100	29 ± 9

P < .05 was determined for F.IX-R338A and hemophilia B/CD 4 mice compared with all other groups. Data presented are mean values ± SEM.

NA indicates not applicable; UD, undetectable.

ANOVA with Tukey-Kramer multiple comparison test was used to compare all individual groups

†

Endogenous murine F.IX⁴⁸

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