Dog AAV Serotype Route Vector genome per kg Vector dose
per site
Transient
Immunosuppression
MaximumcF.IX level
(ng/ml)
Anti-cF.IX
Bethesda Unit
B45 AAV-2 IM 1.3 x 1011 4.1 x 1010 no 2.6 none
B48 AAV-2 IM 3.4 x1012 1.1 x 1012 no 40 none
D32 AAV-2 IM 5.6 x 1012 2.0 x 1012 yes 40 none
D31 AAV-2 IM 8.5 x 1012 2.0 x 1012 no 39 none
B85 AAV-2 IM 8.5 x 1012 2.1 x 1012 no 70 6.8 BU, transient
B14 AAV-2 IM 1.1 x 1013 1.2 x 1013 no 30 ≤ 24.5
E35 AAV-1 IM 1.0 x 1012 5 x 1011 no 87* 8-15 B.U.
E57 AAV-1 IM 2.4 x 1011 6.8 x 1010 yes 104 2-8.5 B.U.
D99 AAV-2 ILP 3.7 x1012 N/A yes 730 none
F57 AAV-2 ILP 1.7 x1012 N/A yes 260 none
H08 AAV-2 ILP 3.0 x1012 N/A yes 210 none
E60 AAV-2 ILP 3.9 x1012 N/A no ~500* 22 BU, long-lasting
               
H48/H34/I07 AAV-2 ATVRX 3.0 x1012 N/A yes 76-275 no
J03 AAV-2 ATVRX 3.0 x1012 N/A no 81 no
J62 AAV-2 ATVRX 3.0 x1012 N/A no 120 1.5 BU, transient
               
M13 AAV-6 ATVRX 3.0 x1012 N/A yes 259 no
M20 AAV-6 ATVRX 3.0 x1012 N/A yes 213 no
Immunosuppression consisted of 6 weekly doses of cyclophosmphamide
#Maximum cF.IX levels detected before antibody formation to F.IX
Table 1: Canine FIX expression by AAV-mediated gene transfer to skeletal muscle in hemophilia B dogs.