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Potential of Gene Therapy
When patients talk with Dr. Barry Byrne, director of the Powell Gene Therapy Center, they ask one question over and over: “When can I try gene therapy?” After all, for those facing chronic illnesses — anything from cardiovascular and pulmonary disease to Parkinson’s — gene therapy offers a promising new horizon.
“Patients are following the developments. They’re clamoring for this type of treatment,” Byrne says. “I can’t wait to tell them, ‘Yes, we’re ready to start.’”
Gene therapy’s potential was becoming clear at the University of Florida in the early 1980s. That’s when College of Medicine professors Drs. Ken Berns and Nicholas Muzyczka modified and patented a groundbreaking gene-therapy delivery system using AAV — short for adeno-associated virus. Their vector serves as a vehicle to deliver healthful genes to patients.
Now — before widespread gene-therapy treatment can proceed — scientists are making sure the treatment is safe and effective. In that effort, UF is again leading the way. The university’s National Gene Vector Laboratory Toxicology Core Center, for instance, is one of just two such facilities nationwide that administer tests for many gene-therapy studies. And UF is also conducting clinical trials for inherited eye disease and Pompe disease, a hereditary heart condition that is usually fatal within the first year. In the Pompe disease trial, the corrective gene is packaged in a type of AAV that collects in heart tissue, allowing the treatment to be delivered by a single injection into a vein, rather than directly into the heart. Byrne hopes that a single treatment will provide patients with lasting benefits.
“One of the key findings from the work done here is that these are long-lasting therapies,” Byrne says. “That’s the neat thing about molecular medicine that’s different from conventional drug therapy.”
Byrne is looking forward to the day that he can offer gene therapy to those with conditions that can’t be cured by traditional medicine.
“It’s an exciting time, there’s no doubt about it,” he says.
“When you can take a problem that seems unbeatable and offer a novel therapy to combat it, that’s exciting.”
