Barry Byrne, M.D., Ph.D.

Barry ByrneDivision

Child Health Research Institute

Academic Title

Director, UF Powell Gene Therapy Center
Professor, Pediatrics and Molecular Genetics & Microbiology
Associate Chair, Pediatrics



Congenital Heart Center
Molecular Genetics


  • Postdoctoral Fellow, Johns Hopkins University
  • Intern and Resident, Johns Hopkins Hospital
  • M.D., University of Illinois
  • Ph.D., University of Illinois
  • B.S., Denison University

Research Overview

Barry Byrne’s research is aimed at understanding types of inherited muscle disease, which lead to heart and skeletal muscle dysfunction. Current research focus is aimed to help those affected by Pompe Disease, an autosomal recessive lysosomal storage disease, and Barth Syndrome, an chromosomal X-linked defect.

Byrne and colleagues are taking two approaches to the problem. The first approach is vector replacement using rAAV, a recombinant virus vector constructed from adeno associated virus (AAV). Second, the Byrne lab is using protein-replacement therapy in an effort to treat deficiencies caused by genetic disorders with specific supplementation.


Dr. Barry J. Byrne is a clinician scientist interested in a variety of rare diseases, with specific attention to developing therapies for inherited muscle disease. As a pediatric cardiologist, his focus is on conditions that lead to skeletal muscle weakness, cardiac dysfunction and respiratory dysfunction. His research team has made significant contributions to the understanding and treatment of Pompe disease, a type of muscular dystrophy resulting from abnormal glycogen accumulation in the muscle. His current research has focused on developing new therapies using the missing cellular protein or the corrective gene to restore muscle function in Pompe and other inherited myopathies.

Dr. Barry Byrne is the Associate Chair of Pediatrics and Director of the Powell Gene Therapy Center at the University of Florida. After obtaining a B.S. degree in Chemistry from Denison University, he pursued his medical education, as well as a Ph.D. in Microbiology and Immunology, at the University of Illinois. He completed his pediatric residency, cardiology fellowship training and post-doctoral training in Biological Chemistry at Johns Hopkins University. Joining the University of Florida in 1997, he has served in a variety of clinical, research and educational roles, and is now the Earl and Christy Powell University Chair in Genetics.


Key Publications

Found at Pubmed: under “Byrne BJ”[Author]