Byrne believes gene therapy is a way to augment the current treatment for Pompe patients, which involves intravenous infusions to replace the missing GAA enzyme.

Efforts to find a treatment for Pompe disease, some of them based on the work of UF's gene therapy and pediatric doctors, are depicted in Pulitzer Prize-winning reporter Geeta Anand's book "The Cure."

The book became the basis of the film "Extraordinary Measures" starring Harrison Ford and Brendan Fraser, which opened last weekend. Byrne provided technical advice to the producers during filming and has a brief moment as an extra in a scene with Fraser.

The film depicts the true story of John and Aileen Crowley, whose two youngest children were diagnosed with Pompe disease. Faced with no options, John Crowley quit his job as a marketing executive and started a biotechnology company, Novazyme Pharmaceuticals Inc., which was eventually purchased by Genzyme Corp.

A cure has yet to be found, but Byrne is hopeful that advances can be made to improve the quality of life for patients and their families.

"We think the gene therapy aspect will work alongside the traditional treatment," said Byrne, who is also a member of the UF Genetics Institute. "In chemotherapy, a combination of treatments is used to benefit the patient, and we hope this will work in the same way."

Source: University of Florida