Melissa Spencer PhD

Dr. Melissa J. Spencer, PhD is a Professor of Neurology at the University of California, Los Angeles (UCLA) and a leading researcher in neuromuscular diseases and gene therapy. She serves as Director of the Neuromuscular Division and Co-Director of the Center for Duchenne Muscular Dystrophy at UCLA, where she leads translational research initiatives aimed at improving treatments for muscle disorders.

Dr. Spencer’s research focuses on understanding the molecular and cellular mechanisms underlying inherited neuromuscular conditions such as Duchenne muscular dystrophy and limb-girdle muscular dystrophy. Her work explores pathways that regulate muscle health and disease progression, intending to develop innovative therapies.

A major area of her research involves advancing gene therapy approaches, including CRISPR-based gene editing and adeno-associated viral (AAV) vector technologies, to deliver therapeutic genes to muscle and stem cells. She also investigates immune responses to gene therapies and strategies to improve delivery efficiency and safety.

With over 25 years of experience in translational muscle research, Dr. Spencer has led numerous NIH-funded studies and contributed significantly to the development of novel therapeutic strategies for neuromuscular diseases. Her work bridges basic science and clinical application, aiming to bring effective, long-term treatments to patients with genetic muscle disorders.