Table 1. Hepatic gene transfer... | American Society of Hematology

Table 1.

Hepatic gene transfer efficiency by different serotypes of AAV-cFVIII vectors in hemophilia A mice

			cFVIII DNA copy/diploid genome
AAV serotype	Vector dose, vg/mouse	No. of mice analyzed	Southern blot, mean ± SEM	QPCR, mean ± SEM
5	3 × 10¹¹	4	0.08 ± 0.02	ND
5	6 × 10¹¹	4	0.40 ± 0.20	ND
2	3 × 10¹¹	10	1.24 ± 0.25	1.73 ± 1.37
2	6 × 10¹¹	8	3.01 ± 0.41	3.86 ± 1.36
6	1 × 10¹¹	3	0.74 ± 0.14	1.11 ± 0.37
6	3 × 10¹¹	10	4.88 ± 0.58	5.78 ± 2.29
6	6 × 10¹¹	4	9.79 ± 1.73	14.97 ± 6.57
8	1 × 10¹¹	6	1.95 ± 0.26	2.21 ± 0.68
8	3 × 10¹¹	6	8.73 ± 0.81	9.68 ± 3.59

			cFVIII DNA copy/diploid genome
AAV serotype	Vector dose, vg/mouse	No. of mice analyzed	Southern blot, mean ± SEM	QPCR, mean ± SEM
5	3 × 10¹¹	4	0.08 ± 0.02	ND
5	6 × 10¹¹	4	0.40 ± 0.20	ND
2	3 × 10¹¹	10	1.24 ± 0.25	1.73 ± 1.37
2	6 × 10¹¹	8	3.01 ± 0.41	3.86 ± 1.36
6	1 × 10¹¹	3	0.74 ± 0.14	1.11 ± 0.37
6	3 × 10¹¹	10	4.88 ± 0.58	5.78 ± 2.29
6	6 × 10¹¹	4	9.79 ± 1.73	14.97 ± 6.57
8	1 × 10¹¹	6	1.95 ± 0.26	2.21 ± 0.68
8	3 × 10¹¹	6	8.73 ± 0.81	9.68 ± 3.59

ND indicates not determined.