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Barry Byrne, MD, PhD

Basic Science/Clinical Research Mentor

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

Dr. Byrne is an Endowed Professor and Associate Chair for Research in the Department of Pediatrics and the Director of the Powell Gene Therapy Center. He is a clinician scientist who is studying a variety of rare diseases with the specific goal of developing therapies for inherited muscle disease. His group has made significant contributions to the understanding and treatment of Pompe disease, a type of neuromuscular disease due to glycogen storage in motor units. His research team has been developing new therapies using AAV-mediated gene therapy to restore muscle function in Pompe and other inherited myopathies, including the most common form Duchenne Muscular Dystrophy. He has sustained NIH funding for over 20 years and is currently the PI of a Program Project developing new therapies in cardiopulmonary disease. He has extensive experience with clinical trials and in specific clinical trials that focus on innovative treatment strategies. He is a pioneer in implementing complementary treatment strategies combining gene therapy and rehabilitation. Dr. Byrne serves as the editor of two journals and is the previous chair of the NIH study section, Skeletal Muscle and Exercise Physiology. As a mentor, he has provided training to pre-doctoral, medical and postdoctoral fellows with over 20 trainees in his career. He has mentored both clinical and basic science young investigators, including several former NMPT trainees and RRCD Scholars. He has trained and continues to train NMPT Trainees with both clinical and basic science backgrounds. Dr. Byrne has been a senior NMPT faculty mentor and has served on the Internal Steering Committee since the inception of the program.