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’s disease (PD).

The Powell Gene Therapy Center has Been a Pioneer in Several Ways:

Birth Place of AAV Vectors

Year Event
1982 AAV genome Cloned – Muzyczaka/Samulski
1983 First AAV vector gene transfer – Muzyczaka/Samulski
1990 – 2000 Proof-of-concept studies & Production Protocols
2001 PGTC founded – Flotte/Byrne
2003 a1AAT Trial – Flotte/Byrne
2005 LCA Trial – Hauswirth/Byrne
2006 a1AAT Trial – Flotte/Byrne
2009 Pompe Trial – Byrne
2010 MerTK Trial – Hauswith
2011 LHON Trial – Guy
2012 AADC Trial – Hwu
2017 Canavan Trial – Byrne
2019 Pompe Trial – Byrne
2019 FA Trial – Byrne/Corti
Birth Place of AAV Vectors