Powell Gene Therapy Center (PGTC)

The PGTC Center, directed by Dr. Barry Byrne, was founded in 1996. Its primary mission is to merge molecular genetics research and health care delivery by developing new therapeutic strategies for the treatment of human diseases that involve gene transfer. The PGTC was recently named one of only two National Gene Vector Laboratory (NGVL) Toxicology Centers by the NCRR and was also selected by the NCRR as the site for 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 to allow for greater sharing of data among various centers. The PGTC has been awarded 4 NIH program project grants totaling approximately $25 M to pursue gene therapy for diseases of the heart, lungs, and liver and has been designated as a Diabetes Gene Therapy Center by the Juvenile Diabetes Research Foundation. The PGTC has pioneered several advances relevant to gene delivery including discovery of the unique life cycle of AAV; first use of AAV to deliver genes to cells in culture; first use of AAV in animals and humans; first use of AAV in models of muscle and heart disease; first use of AAV to reverse blindness in dogs with a genetic disease; active clinical trials in CF patients since

1996. Several of the Lead mentors (Krista Vandenborne. Glenn Walter, Paul Reier, Dave Fuller) have ongoing collaborations with the Powell Gene Therapy Center and are combining gene transfer and rehabilitation strategies to improve function. Areas of particular expertise are Duchenne Muscular Dystrophy, Glycogen Storage Disease and Spinal Cord Injury.

The Vector Core laboratory manufactures research-grade gene transfer vectors for use in pre-clinical investigations, while the Human Applications Lab manufactures gene transfer vectors under cGMP for use in human clinical trials. The Human Applications Laboratory is a state of the art GMP manufacturing facility that occupies 2200 sq ft in the Mc Knight Brain Institute. The unit includes an 1800 sq ft BL2/BL3 GMP facility, a 300 sq ft Quality Control laboratory and storage facilities for receipt and control of incoming materials. The GMP manufacturing facility has two suites with 2 production modules in each module which operate on a campaign basis. The first suite deals with Cell Processing and the other is used for Viral Vector Production.