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Jeffrey S. Chamberlain, PhD

Department of Neurology, Biochemistry, and Medical Genetics
University of Washington School of Medicine
Health Sciences Building, K-253
Seattle, Washington, United States

Dr. Jeffrey Chamberlain is a neurologist with expertise in muscular dystrophies. Currently, he is working as professor in the departments of Neurology, Biochemistry, and division of Medical Genetics at the University of Washington School of Medicine.

His research focuses on presenting better understanding of the molecular basis of the pathophysiology of the neuromuscular diseases along with developing gene and cell therapies that will correct and treat the muscular dystrophies and other muscular disorders. Major targets for his therapy research include Duchenne muscular dystrophy and Limb-girdle muscular dystrophy type 2I. Chamberlain and his associates are studying the structure and function of the dystrophin gene, as well as developing viral vectors to deliver this or other genes to muscle cells for possible gene therapy.

He authored a book with Thomas A. Rando titled “Duchenne Muscular Dystrophy: Advances in Therapeutics (Neurological Disease and Therapy)”.


Representative Publications:

AAV6 Vector Production and Purification for Muscle Gene Therapy

Progress toward Gene Therapy for Duchenne Muscular Dystrophy

Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy

Prosurvival Factors Improve Functional Engraftment of Myogenically Converted Dermal Cells into Dystrophic Skeletal Muscle

Gene Therapy for Duchenne muscular dystrophy