A Pioneer in Gene Therapy
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).
The Vector Core manufactures research-grade gene transfer vectors for use in pre-clinical investigations.
The Human Applications Laboratory manufactures gene transfer vectors under cGMP for use in human clinical trials.
The Toxicology Core is responsible for conducting animal and in vitro toxicology studies that the FDA determines are needed before a particular clinical gene transfer trial can begin.
The mission of the Process and Development Laboratory of PGTC (PD) is to develop methods for large-scale manufacturing of rAAV stocks in a GMP-translatable manner.