Table 2.

Summary of results of transvenular delivery of AAV6-cFIX at 3 × 10¹² vg/kg gene transfer to skeletal muscle of severe hemophilia B dogs

HB dog	cFIX variant	Plateau cFIX expression*			Spontaneous bleeds/mo
		Activity, %	Antigen, %	Specific activity	Before	After
M13†	WT (R338)	5.2 ± 3	5.5 ± 0.8	1.0		0/12
M20†	WT (R338)	6.2 ± 3	4.1 ± 0.2	1.5		0/12
N40	R338I	0.95 ± 0.01	0.22 ± 0.05	4.4	6/45	2/9
M55‡	R338L	8.7 ± 3	1.4 ± 0.3	7.4	0/7	0/101
M59‡	R338L	3.5 ± 1	0.52 ± 0.2	7.7	0/6	0/101
N07‡	R338L	5.7 ± 2	0.60 ± 0.3	10.6	2/6	0/79

^*Mean values after plateau ± standard deviation.

^†Previously reported.¹²

^‡Previously reported.^14,15