Scott Harper, PhD

Join radio host Tim Hollenback on the show this month is FSHD research rock star Scott Harper, PhD, a leader in the effort to develop gene therapy. Harper is principal investigator at the Center for Gene Therapy at the Research Institute at Nationwide Children’s Hospital, and associate professor of pediatrics at the Ohio State University College of Medicine, Columbus, Ohio. He is a molecular biologist who has spent his career working to develop gene therapy approaches for muscular dystrophies. As a PhD student at the University of Michigan (1997-2002), he and colleagues created “micro-dystrophin” gene therapy to treat Duchenne Muscular Dystrophy (DMD) in mice. Micro-dystrophin gene therapy is now being independently tested in boys with DMD. The lessons learned in DMD can now be applied to develop gene therapies for FSHD, and this has been a main focus of the Harper lab during the past decade. Dr. Harper was selected as the Outstanding New Investigator by the American Society of Gene and Cell Therapy in 2014.