Percutaneous Transendocardial Delivery of Self-Complementary Adeno-Associated Virus 6 in the Canine
Achieving efficient cardiac gene transfer in a large animal model has proven to be technically challenging. Prior strategies have employed cardio-pulmonary bypass or dual catheterization with the aid of vasodilators to deliver vectors, such as adenovirus, adeno-associated virus (AAV) or plasmid DNA. While single-stranded AAV vectors have shown the greatest promise, they suffer from delayed expression, which might be circumvented by using self-complementary vectors. We have recently optimized a cardiac gene transfer protocol in the canine using a percutaneous transendocardial injection catheter to deliver an AAV vector under fluoroscopic guidance. Percutaneous transendocardial injection of self-complementary AAV (scAAV)-6 is a safe, effective method for achieving efficient cardiac gene transfer to approximately 60% of the myocardium.
Key wordsGene therapy Cardiac Canine model AAV Vector delivery Adeno-associated virus
This work was funded by a grant from the National Heart, Lung, and Blood Institute (P01-HL059407) (to H.L.S.), the Parent Project Muscular Dystrophy (to H.L.S.), and by T32-HL-007748 (to L.T.B).
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