Jeffrey Chamberlain

Dr. Chamberlain is a geneticist with expertise in the muscular dystrophies.  He is a professor of neurology, medicine and biochemistry at the University of Washington, director of the Wellstone Muscular Dystrophy Specialized Research Center, and the McCaw Endowed Chair in Muscular Dystrophy. His research focuses on understanding mechanisms leading to, and developing treatments for, the muscular dystrophies, with a focus on gene therapy. His lab invented ‘micro-dystrophin’ and discovered that some serotypes of AAV can be used for systemic gene delivery to muscle. This work led to the strategy of using AAV vectors to treat Duhenne muscular dystrophy via systemic delivery of microdystrophin cassettes. Several of his vectors are currently being tested in human clinical trials. Dr. Chamberlain has served on numerous US and European study sections, as well as advisory boards for NIH, the FDA, the Muscular Dystrophy Association and several Biotech companies focused on muscle therapeutics. He is the current  President of the American Society for Gene and Cell Therap.