Scott Q. Harper

Scott Q. Harper's research focuses on developing gene therapies for inherited neuromuscular and neurological disorders, including facioscapulohumeral muscular dystrophy (FSHD), Charcot-Marie-Tooth disease type 1A (CMT1A), and genetic epilepsies caused by specific gene mutations (such as GRIN2D and DNM1). His work often involves designing RNA interference (RNAi) and adeno-associated virus (AAV)-based tools to silence disease-causing genes or replace their function, and testing these therapies in animal models and cell-based systems. He also studies disease mechanisms, such as how the DUX4 protein contributes to muscle degeneration in FSHD, and explores biomarkers and single-cell analysis techniques to better understand muscle disease progression.

Publication output has remained relatively steady but modest over the past decade, fluctuating between roughly 2 and 7 papers per year without a clear long-term increase or decline.

Generated by claude-sonnet-5 from public bibliographic data · Jul 20, 2026