Dr. Barry J Byrne is currently Director of UF Powell Gene Therapy Center at the same time Professor of Pediatrics and Molecular Genetics & Microbiology and Associate Chair of Pediatrics at University of Florida. Dr. Barry J Byrne's laboratory is focused on understanding the pathophysiology of inherited cardioskeletal myopathies and therapeutic strategies using gene therapy. Specifically, they have developed novel gene therapies for neuromuscular diseases such as Pompe disease. The disease is characteristic of many muscle diseases which lead to skeletal and cardiac muscle weakness. Dr. Barry J Byrne has used AAV vectors to achieve sustained correction of the gene deficiency in Pompe disease models leading to restoration of contractile and metabolic function of striated muscle and motor nerves. Recently, Dr. Barry J Byrne has incorporated novel combinations of AAV serotypes and/or capsid modifications with tissue-specific promoters to direct gene transduction and expression of the therapeutic transgene. Dr. Barry J Byrne approach (AAV) is now being evaluated in a human clinical trial investigating the safety and efficacy of direct gene delivery to the diaphragm.

Dr. Barry J Byrne's research interests includes: Adeno-associated virus mediated gene transfer in the cardiovascular system and striated muscle, Cell therapy in cardiovascular disease, Gene therapy for muscular dystrophy (Pompe disease and DMD), Mechanisms of allograft tolerance in cardiac transplantation and Cardiac development.