Developing new therapeutic strategies for the treatment of human diseases
The Powell Gene Therapy Center (PGTC) at the University of Florida has been instrumental in the development of newer, safer agents for the delivery of therapeutic genes to patients with genetic diseases, such as cystic fibrosis (CF) and alpha 1-antitrypsin deficiency (AAT-D). New programs will extend the use of the harmless virus, called AAV, that is used as a carrier for therapeutic genes to the study of other genetic disorders such as congenital Leber’s amaurosis (CLA), a genetic cause of blindness, and Pompe’s disease, a genetic disease characterized by heart failure and muscular dystrophy. It may also be applied to important diseases of the central nervous system, such as Parkinson disease (PD).
The Powell Gene Therapy Center has been a pioneer in several ways:
- Discovery of the unique life cycle of AAV by Dr. Kenneth Berns
- First use of AAV to deliver genes to cells in culture by Dr. Nicholas Muzyczka
- First use of AAV in animals and humans by Dr. Terry Flotte
- First use of AAV in models of muscle and heart disease by Dr. Barry Byrne
- First use of AAV to reverse blindness in dogs with a genetic disease by Dr. Bill Hauswirth