|
1
|
Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.
|
Blood
|
2010
|
1.50
|
|
2
|
Efficacy and safety of long-term prophylaxis in severe hemophilia A dogs following liver gene therapy using AAV vectors.
|
Mol Ther
|
2010
|
1.38
|
|
3
|
Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs.
|
Mol Ther
|
2010
|
1.35
|
|
4
|
Hyperactive sleeping beauty transposase enables persistent phenotypic correction in mice and a canine model for hemophilia B.
|
Mol Ther
|
2010
|
1.00
|