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).

manufacturing labs


Vector Core Lab

The Vector Core manufactures research-grade gene transfer vectors for use in pre-clinical investigations.

Vector Core


Human Applications Lab

The Human Applications Laboratory manufactures gene transfer vectors under cGMP for use in human clinical trials.

Human Applications


Toxicology Core

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.

Toxicology Core


Process & Development Lab

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.

Process Development & Quality Control

gene therapy news

What’s Holding Back Gene Therapy?

Category: News & Events

Gene therapy is finally poised to revolutionize the treatment of many diseases, but the nascent field is facing challenges — notably in…

Two test tubes sitting on black counter top

Written in our DNA

Category: News & Events

UF researchers’ groundbreaking work from 1980s leads to new treatment for debilitating neuromuscular disease

close up of baby with nasal cannula

Experts Barry Byrne, Jerry Mendell Lead NORD…

Category: News & Events
Barry Byrne, MD, PhD