What is Pompe Disease? Featured
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Barry J. Byrne, M.D., Ph.D.
Healing and Battling Disease
Dr. Barry Byrne, Director of the Powell Gene Therapy Center at the University of Florida joins the show to explain and discuss a rare genetic disease called Pompe.
Barry J. Byrne, M.D., Ph.D. is a Professor of Pediatrics and Molecular Genetics and Microbiology, College of Medicine, Department of Pediatrics; Molecular Genetics and Microbiology; Director of the Powell Gene Therapy Center at the University of Florida. Dr. Byrne’s laboratory is focused on molecular approaches to diagnosis and treatment of heart failure in infants and children. Therapeutic approaches rely on AAV-mediated gene therapy. As a model system, they are focusing on a form of heart failure due to glycogen storage disease. Additional studies are focused on the use of AAV vectors for gene therapy targeted to striated muscle and liver for the production of other therapeutic proteins. These projects include treatment of glycogen storage disease type I, hemophilia, and other heart failure models. Additional projects in the cardiovascular area are related to the inhibition of thrombosis for vascular grafts, and the development of graft-specific immune suppression by expression of immunoregulatory proteins in cardiac allografts. The use of AAV vectors in conjunction with stem cells is being tested in the context of tissue regeneration in cardiomyopathy with the use of autologous stem cells, which have cardiomyogenic potential. These programs are supported by the AHA, MDA, NIH (NHLBI, NIDDK, and NCRR).