Table 1 Summary of results in hemophilia... | American Society of Hematology

Table 1

Summary of results in hemophilia B dogs after skeletal muscle delivery of AAV-canine FIX R338L

Dog	Age/sex/weight	Plateau cFIX expression		Ratio activity/antigen*	WBCT,† min	Inhibitors	IFN-γ ELISpot
Dog	Age/sex/weight	Activity, %	Antigen, %	Ratio activity/antigen*	WBCT,† min	Inhibitors	AAV	cFIX	338 peptides
M55	7 mo/male/23 kg	8	1.5	8.58 ± 0.46	11.4 ± 0.7	ND	—	—	—
M59	7 mo/male/16.5 kg	3.5	0.35	9.24 ± 0.44	13.7 ± 0.7	ND	ND	ND	ND
N07	6 mo/male/13 kg	5.5	0.65	9.0 ± 0.37	14.4 ± 0.7	ND	ND	ND	ND

Dog	Age/sex/weight	Plateau cFIX expression		Ratio activity/antigen*	WBCT,† min	Inhibitors	IFN-γ ELISpot
Dog	Age/sex/weight	Activity, %	Antigen, %	Ratio activity/antigen*	WBCT,† min	Inhibitors	AAV	cFIX	338 peptides
M55	7 mo/male/23 kg	8	1.5	8.58 ± 0.46	11.4 ± 0.7	ND	—	—	—
M59	7 mo/male/16.5 kg	3.5	0.35	9.24 ± 0.44	13.7 ± 0.7	ND	ND	ND	ND
N07	6 mo/male/13 kg	5.5	0.65	9.0 ± 0.37	14.4 ± 0.7	ND	ND	ND	ND

ND indicates not detected; and —, not done.

Average ratio of activity/antigen ± SE from all time points.

Whole blood clotting time: average values ± SE (normal values, 8-12 minutes).