Nicholas Muzyczka, Ph.D.

Eminent Scholar of Molecular Genetics and Microbiology

College of Medicine

2000 Awardee

A virus? Helpful? Most people wouldn’t think so. But Nicholas Muzyczka is working to prove that sometimes viruses can heal. Muzyczka is widely acknowledged as the world leader in the development of the adeno-associated virus (AAV) as a gene therapy transmitter.

AAV, a human virus that causes no perceptible illness, may provide a vehicle for delivering copies of good genes to patients suffering from hereditary diseases such as cystic fibrosis. “In the simplest cases, the patient is missing a normal gene,” Muzyczka explains. “What [AAV] delivers would correct the disease; AAV itself just disappears… after it delivers the payload.”

Muzyczka first focused on AAV in the mid-1980s while searching for a device to safely and effectively transmit corrective genes. Now, researchers are in a position to clinically test what has been called the “best human gene therapy vector in a wide variety of genetic diseases.”

The UF laboratory Muzyczka directs was the first to show that AAV could be used for gene transfer. The researchers are currently collaborating with a number of other laboratories to gather information about the behavior of AAV in primary cells and whole animals, an area that has been mostly unexplored. They are also focusing on the improvement of current growth methods for manipulated AAV viruses, and are busy formulating the AAV delivery system. Muzyczka and his colleagues at UF were awarded a patent in 1992 for the use of AAV transduction vectors in their work.

Muzyczka has been honored numerous times for his efforts, but he counts his directorship of UF’s Gene Therapy Center as one of his greatest accomplishments. Since becoming director in 1994, he has built the center into one of the top facilities in the world. “[Serving as the first director] allowed me the opportunity to hire a very talented group of people that hopefully will find a way to use the AAV tool to cure someone.”