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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

Contact Information (Research team)


Powell Gene Therapy Center
Powell Center for Rare Disease Research and Therapy
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 Byrne is a clinician scientist who is studying 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 and problems in heart and respiratory function. His group has made significant contributions to the understanding and treatment of Pompe disease, which a type of muscular dystrophy due to abnormal glycogen in the muscle. The research team has been developing new therapies using the missing cellular protein or the corrective gene to restore muscle function in Pompe and other inherited myopathies.

Dr. Byrne is the Associate Chair of Pediatrics and Director of the University of Florida Powell Center for Rare Disease Research.

He obtained his B.S. degree from Denison University, his M.D. and Ph.D. from the University of Illinois and completed his Pediatrics residency, cardiology fellowship training and post-doctoral training in Biological Chemistry at the Johns Hopkins Hospital. He joined the University of Florida in 1997 and is now the Earl and Christy Powell University Chair in Genetics.

Key Publications

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