Extraordinary medicine

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When most people hear the word “virus,” they think runny nose, fever, and maybe an upset stomach. But Barry Byrne ’78 is flipping the script by taking the tiny pathogens and making them work for us, instead of against us.

Byrne is professor of pediatrics and molecular genetics at the University of Florida, where he is using the adeno-associated virus (AAV) to treat Pompe disease, a form of muscular dystrophy. By taking all the genetic code out of the virus, he can then insert new, therapeutic genes, which carry corrective packages into the patient’s cells. AAV is particularly useful because it is a virus that causes no known human disease.

Using this technique, Byrne has corrected Pompe disease in mice and is now working with the FDA to begin human clinical trials.

“I hope we see at time when this strategy can be seen by a larger number of patients rather than just those in clinical trials,” Byrne explained. “I think we are within a reasonable time frame of that happening,”

Byrne’s research has even caught the eye of Hollywood. Extraordinary Measures, a 2010 feature film starring Brendan Fraser and Harrison Ford, follows a father’s determined search for a treatment for Pompe. Byrne worked as a clinical investigator during much of the laboratory drama that the film, which was inspired by a true story, depicts.

His knowledge of Pompe and involvement in the lab led Byrne to one of the film’s locations in Portland, Ore., where he had a chance to meet the actors, as well as Michael Shamberg, the producer. While there, Byrne even appeared in a brief cameo in a scene at the beginning of the movie.

Along with his B.S. in chemistry from Denison, Byrne holds a Ph.D and an M.D. from the University of Illinois. He also was a post-doctoral fellow at Johns Hopkins University, where he did his medical residency. Watch the video above for an in-depth look at his innovative treatment, as well as a firsthand account of his experience on set.

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5:38 AM August 5, 2011

joan maccracken, MD wrote:

Extraordinary work. No longer just science fiction and certainly gene manipulation at its best.

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