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First Gene Therapy Human Trial For Muscular Dystrophy In Progress

zing it, while retaining the crucial elements of its set of DNA instructions, has been among the greatest challenges to the gene therapy field.

The therapy was made possible by the pioneering research in AAV by Dr. Richard Jude Samulski, professor of pharmacology and director of the Gene Therapy Center at UNC, and Dr. Xiao Xiao, a former UNC postdoctoral researcher in Samulski's laboratory now with the University of Pittsburgh Human Gene Therapy Center and associate professor of orthopedic surgery.

Samulski has long pioneered methodologies for making viruses deliver genes. As a graduate student at the University of Florida in the early 1980s, his thesis project was developing the AAV as a vector for therapeutic genes. This work eventually led to isolation of type-2 AAV, which has been used for gene therapy trials in cystic fibrosis and in several other settings, Samulski said.

At Pittsburgh, Xiao had developed a miniaturized version of the gene for dystrophin, the muscle protein needed by people with DMD. Eager to test it in a vector, he contacted his former mentor.

"The dystrophy gene is like a long picket fence. It's the largest gene in the human body, occupying 1 percent of the X chromosome," Samulski said. "Xiao Xiao began removing pegs, or pickets, from the fence, making it smaller and smaller but kept testing to see if it would still perform its function," Samulski said.

Xiao then began looking for a way he might move his minidystrophin gene forward clinically.

"We had just finished demonstrating that we could make clinical-grade virus for another genetic disorder called Canavan's disease, an enzyme deficiency disorder," Samulski said. "We were the first academic institution to ever make FDA-certified AAV vectors to go into the brains of children with Canavan's disease. So we had cut our teeth and had a bit of a track record by 2002, and that's when Xiao approached me."

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