The Powell Gene Therapy Center (PGTC) at the University of Florida (UF) 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).
PGTC has been a pioneer in several ways:
- Discovery of the unique life cycle of AAV (Dr. Kenneth Berns)
- First use of AAV to deliver genes to cells in culture (Dr. Nicholas Muzyczka)
- First use of AAV in animals and humans (Dr. Terry Flotte)
- First use of AAV to in models of muscle and heart disease (Dr. Barry Byrne)
- First use of AAV to reverse blindness in dogs with a genetic disease (Dr. Bill Hauswirth)
- Active clinical trials in CF patients since 1996, including two that are currently enrolling patients (Dr. Terry Flotte)
- A clinical trial in AAT deficiency (genetic emphysema) to begin in early 2004 (Drs. Terry Flotte and Mark Brantly)
- The PGTC has competed successfully for federal funding in numerous arenas:
UF has just been named one of only two National Gene Vector Laboratory (NGVL) Toxicology Centers by the National Center for Research Resources, a Division of NIH
- UF was also selected by the NGVL as the site production of the National Reference Standard Stock of AAV, to set the standard for the potency and purity of these key gene transfer reagents, and allow for greater sharing of data among various centers.
- The PGTC has been awarded 4 separate NIH program project grants totally approximately $21 million of funding to pursue gene therapy for diseases of the heart, lungs, and liver
- The PGTC has been designated as a Diabetes Gene Therapy Center by the Juvenile Diabetes Research Foundation
- The Alpha One Foundation, CF Foundation, American Heart Association, Muscular Dystrophy Association and a number of other disease foundations have partnered with UF to move gene therapy forward with their diseases of interest.
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