Scientific Advisory Board

David A. Weinstein, M.D., M.M.Sc. Director, Glycogen Storage Disease Program; Professor, University of Florida College of Medicine | Web Page

Following his graduation from Trinity College (CT) in 1990 and Harvard Medical School in 1994, Dr. David Weinstein completed a residency, chief residency, and fellowship in pediatric endocrinology at Children's Hospital Boston. He subsequently obtained a Masters in clinical investigation from Harvard and MIT, and became Director of the Glycogen Storage Disease Program at Children's Hospital Boston. In 2005, Dr. Weinstein moved to the University of Florida where he directs the Glycogen Storage Disease Program and is a tenured Associate Professor. Dr. Weinstein follows over 300 patients with glycogen storage disease, and he has published over 40 articles and 17 textbook chapters on this topic. He is a former Jan Albrecht Award winner from the American Association for the Study of Liver Diseases, and he was awarded the George A. Sacher Award from the Gerontological Society of America. Dr. Weinstein has served on both the Board of Directors and the Scientific Advisory Committee for the Association for Glycogen Storage Disease. He also served on the international consensus panels assembled to create clinical guidelines for management of GSD types Ia, Ib, and III.

Thomas Conlon, Ph.D., Director of PGTC Toxicology Center, Assistant Professor in Pediatrics, University of Florida College of Medicine | Web Page

As the Director of the National Gene Vector Laboratory (NGVL) Toxicology Center at the University of Florida, Dr. Conlon facilitates efficient, cost-effective, and rigorous preclinical testing of gene therapy vectors, with a special emphasis on recombinant adeno-associated virus (rAAV) vectors. His team performs preclinical studies on subjects ranging from mice to swine and primates that have supported the successful Investigational New Drug submissions for phase I trials of rAAV in Alpha-1 Antitrypsin deficiency, congestive heart failure (CHF) and Leber Congenital Amaurosis (RPE65). UF has taken the lead in sharing those preclinical data with the NGVL by depositing data in the rAAV drug master file (DMF). The development of centralized core facilities for vector production, animal toxicology, pathology, and biostatistics/bioinformatics has allowed these procedures to proceed more efficiently.Dr. Conlon is a native Texan with roots in Dallas. Having received his BS in Genetics from the Texas A&M University under the tutelage of James R. Wild, PhD, Thomas moved to Gainesville in order to begin his graduate education in the Interdisciplinary Program in Biomedical Sciences (IDP) with a concentration in genetics. His thesis entitled Hepatocyte Transduction by Recombinant Adeno-associated Virus as an Approach to Gene Therapy for Alpha-1 Antitrypsin Deficiency and Fatty Acid Oxidation Disorders was earned under the direction of Dr. Terry Flotte in 2004. Following graduation, he took the position of Director of the National Gene Vector Laboratory where he has served since as well as being a faculty member in the Department of Pediatrics.

Dwight D. Koeberl, MD, PhD., Associate Professor of Pediatrics, Duke University School of Medicine | Web Page

Dwight Koeberl, MD,PhD is an Associate Professor in the Department of Pediatrics/Division of Medical Genetics at Duke University Medical School, with a secondary appointment in Molecular Genetics and Microbiology (MGM). He is the Medical Director for both the Mass Specrometry and Glycogen Storage Disease Laboratories.

Dr. Koeberl received a B.A. in Chemistry at Carleton College, and M.D.,Ph.D. in Biochemistry from Mayo Graduate and Medical Schools. He ventured to the University of California in San Francisco for pediatrics residency training, and completed clinical and biochemical genetics fellowship training at the University of Washington in Seattle. He joined the Division of Medical Genetic at Duke on a research track position in 1999, focusing primarily on the development of gene therapy for glycogen storage diseases. Dr. Koeberl has co-authored >60 peer-reviewed publications. Dr Koeberl has served on the Board of Directors for the Association for Glycogen Storage Disease, and co-wrote consensus panels assembled to create clinical guidelines for management of GSD types Ia, Ib, V, and VII. The focus of research in his laboratory at Duke has been gene therapy with viral vectors, including adeno-associated virus (AAV) vectors and adenovirus vectors, for glycogen storage disease type Ia (GSD1A). They have developed gene therapy with a novel AAV vector that: 1) prevented mortality and corrected glycogen storage in the liver; 2) corrected hypoglycemia; and 3) achieved efficacy with 100-fold fewer vector particles. The demonstration of long-term efficacy of gene therapy in the canine model for GSD1A provides the proof-of-concept needed to justify clinical translation of gene therapy in GSD1A.

Barry Byrne, MD, PhD., Director University of Florida Gene Therapy Center; Professor of Pediatrics and Molecular Genetics and Microbiology, University of Florida College of Medicine | Web Page

Dr. Byrne is a Pediatric Cardiologist clinician and Associate chair of the Congenital Heart Center at UF. He is a professor in pediatrics and molecular genetics and is currently the director of the University of Florida Powell Gene Therapy Center. His research is aimed to help those affected by genetic muscular diseases and he is currently the PI on multiple clinical trials for this effort. His primary involvement lies with disorders such as Pompe, Barth, muscular dystrophy, and the associated cardiomyopathy of these diseases. Dr. Byrne obtained his B.S. from Denison University in Ohio, his M.D. and Ph.D. from the University of Illinois, and completed his residency and fellowship training at Johns Hopkins University in Maryland. He was an Assistant Professor at Hopkins after completing his training before coming to the University of Florida in 1997.