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Gene Therapy Technique for Inherited Heart Problems in The Pipeline

with scientists at the California National Primate Research Center at the University of California at Davis showed that AAV-9 easily passes into the heart muscle in primates after a single intravenous injection, and the effects are long lasting. ‘AAV-9 had a particular affinity for heart muscle that AAV-8 didn't,’ Byrne said. ‘Right now AAV-9 seems to be our best solution for heart problems. ‘The primates were done to give us insight into how this could be used in children,’ he said, adding that UF researchers hope to launch a trial in patients with Pompe disease early next year. ‘Many of the inborn errors have their most feared effects early in life, so we're pushing toward being able to do prevent disease as opposed to trying to correct later. We keep trying to get closer and closer to clinical studies that will help patients-that's the real goal.’

R. Jude Samulski, Ph.D., director of the Gene Therapy Center at the University of North Carolina-Chapel Hill, said each viral type is ‘like a FedEx truck that carries a ZIP code that determines where the truck is going to take its payload to.’ ‘Laboratories are engineering new ZIP codes onto these vectors so they can try to control where they go,’ he said. Then they become more like a traditional drug that has these properties and only these properties. We're using templates out in Mother Nature as our blueprint. Once we learn these places where they work, then we can strategically control them and use them for our advantage. It's an exciting time in this area. ‘I equate it to the space program,’ he added. ‘We don't know who Neil Armstrong is yet, who's going to be the one to make this work, but we know we're going to the moon. Everybody engineering things is part of that effort. What Barry has done is more significant than what people imagined. It's close to the Holy Grail when you get it to work in small animals and then you get it to work in large animals. What you have left is to go into humans. It's real
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