Dr. Spencer is currently a Professor of Neurology and a co-director for the Center for Duchenne Muscular Dystrophy at UCLA, which is a translational center that supports both basic and clinical research. In her role as co-director and as Director of the Neuromuscular Division in the Department of Neurology, she leads several efforts pertaining to growth of the center including the Pediatric Neuromuscular Clinic, the organization of the annual retreat, working group meetings and pilot and feasibility program. She has been conducting translational muscle research for over 25 years and has been the PI on both basic and clinical studies, including a P30 Core Center. She was awarded a PECASE in 2001 and has continued to serve in leadership and advisory roles for federal and private agencies in the muscular dystrophy arena and on the UCLA campus.
The research program in the Spencer Lab focuses on understanding normal skeletal muscle biology and on how alterations or defects in normal processes (via genetic mutations) lead to muscle disease. The lab seeks to gain mechanistic insights on skeletal muscle signaling that leads to functional adaptation, stem cell biology, inflammation in normal and disease settings and understanding the pathogenic progression of muscular dystrophies. By gaining insight into normal muscle processes, this knowledge can subsequently be applied towards therapeutic interventions. A key area of interest in the lab is the genetically inherited muscular dystrophies, primarily limb girdle muscular dystrophy and Duchenne. Significant work has been done on LGMD type 2A, due to CAPN3 mutations and this work has led to the key finding that CAPN3 and CAMKII signaling are integral to skeletal muscle remodeling. The lab also focuses on characterizing inflammatory processes that participate in DMD pathophysiology and on developing gene therapeutics for Duchenne muscular dystrophy. In particular, the lab is working with Drs. April Pyle and Huan Meng on development of a CRISPR/Cas9 based gene editing platform and nanoparticle mediated delivery systems.
Dr. Shieh earned his MD and PhD degrees from the Johns Hopkins University of Medicine before completing his Neurology Residency Training at Stanford and his Clinical Neurophysiology Fellowship training at Partners in Boston. He joined the faculty in 2003.
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