Scott Harper

Scott Harper, PhD, is a principal investigator in the Jerry R. Mendell Center for Gene Therapy at the Abigail Wexner Research Institute at Nationwide Children's Hospital and a professor of Pediatrics at The Ohio State University College of Medicine. He serves as a standing member of the NIH Neurological Sciences and Disorders B (NSD-B) study section, Scientific Advisory Board member for the Charcot-Marie-Tooth Association (CMTA), and participates in multiple committees of the American Society of Gene and Cell Therapy (ASGCT). As chief scientific advisor at Armatus Bio, Dr. Harper bridges academic research with clinical translation.

Dr. Harper earned his doctorate in Cellular and Molecular Biology from the University of Michigan Medical School, working with Jeff Chamberlain to develop early micro-dystrophin gene therapies for Duchenne Muscular Dystrophy. His postdoctoral training at the University of Iowa with Beverly Davidson focused on RNAi-based gene therapies for dominant neurodegenerative diseases. Recruited to Nationwide Children's Hospital in 2007 by Jerry Mendell, he established his laboratory focusing on RNAi applications for muscle diseases.

His research centers on developing adeno-associated virus (AAV) based gene therapies for neuromuscular disorders, with significant contributions to FSHD research through DUX4 gene silencing approaches. His work spans muscular dystrophies (FSHD, LGMD1A), peripheral neuropathies (CMT1A, CMT2D, CMT4B3), and dominant epilepsy, emphasizing translational research from bench to bedside. His laboratory's 2020 proof-of-concept study in Molecular Therapy: Nucleic Acids demonstrated DUX4 silencing in human cells, including those from FSHD patients.

Dr. Harper has witnessed the remarkable translation of his early graduate work on micro-dystrophin to clinical application, with therapies based on his constructs now in clinical trials for DMD. His publications reveal a consistent trajectory of innovation in vector design, therapeutic targeting, and overcoming immune responses in gene therapy, with increasing focus on clinical translation in recent years.

• Featured Researcher by Abigail Wexner Research Institute (November 2021)
• Standing member, NIH Neurological Sciences and Disorders B study section
• Scientific Advisory Board, Charcot-Marie-Tooth Association
• Committee member, American Society of Gene and Cell Therapy

Dr. Harper actively collaborates with patient advocacy groups globally, having traveled to Australia and New Zealand to educate FSHD communities. He serves as chief scientific advisor for Armatus Bio, which is licensing his laboratory's technology. Despite administrative responsibilities, he prioritizes maintaining an active laboratory presence, focusing on developing new therapeutic approaches while mentoring the next generation of researchers.