OUR GUEST SPEAKERS
Terence R . Flotte , M . D ., is the executive deputy chancellor and provost of UMass Chan Medical School and dean of the T . H . Chan School of Medicine in Worcester . Dr . Flotte is an internationally celebrated pioneer in human gene therapy . He led the first team to use the adeno-associated virus as a vehicle to deliver corrective genes to targeted sites in the body . Dr . Flotte ’ s current research involves the use of gene therapy for alpha-1 antitrypsin deficiency and Tay-Sachs disease .
David Seo , M . D ., is the senior vice president and chief digital and information officer at the Nicklaus Children ’ s Health System , Miami , Florida . Dr . Seo oversees the strategic initiatives of the Nicklaus Children ’ s Health System and its network of more than 25 satellite locations . His leadership has contributed to remarkable achievements in pediatric rare disease research and therapy via newborn genetic sequencing and pharmacogenomics programs .
Nutan Sharma , M . D ., Ph . D ., is the director of the Collaborative Center for X-linked Dystonia Parkinsonism at the Massachusetts General Hospital , Boston . She is also the director of the Dystonia Clinic at Mass General and associate professor of neurology at Harvard Medical School . Dr . Sharma ’ s research includes the study of single nucleotide polymorphisms in the Tor1A gene , and fMRI studies to evaluate white matter microstructure in dystonia .
Mustafa Tekin , M . D ., chairs the Dr . John T . Macdonald Foundation Department of Human Genetics and is chief of the Division of Clinical and Translational Genetics at the University of Miami Miller School of Medicine . He is also director of the Miami site of the NIH-funded Undiagnosed Diseases Network and the Miami site of the national Network of Rare Diseases ( NORD ) Centers of Excellence , and a member of Florida Rare Disease Advisory Council . His research program is focused on the etiology of rare genetic disorders with an emphasis on hereditary hearing loss .
Barry Byrne , M . D ., Ph . D ., is the associate chair of Pediatrics and director of the Powell Gene Therapy Center at the University of Florida ’ s College of Medicine . Dr . Byrne ’ s research is focused on rare diseases , specifically developing therapies for inherited muscle disease . His research team has made significant contributions to the understanding and treatment of Pompe disease , Duchenne muscular dystrophy and other inherited myopathies .
Philip J . ( P . J .) Brooks , Ph . D ., is the deputy director of the Division of Rare Diseases Research Innovation at the National Institutes of Health ’ s ( NIH ) National Center for Advancing Translational Sciences ( NCATS ) in Rockville , Maryland . He represents NCATS in the NIH ’ s Gene Therapy Working Group and Regenerative Medicine Innovation Project , as well as the International Rare Diseases Research Consortium ( IRDiRC ). Dr . Brooks is also co-coordinator for the NIH Common Fund program on Somatic Cell Genome Editing ( SCGE ), a leader of the Platform Vector Gene Therapy ( PaVe-GT ) pilot project and co-chairs the Bespoke Gene Therapy Consortium .