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 |