Jeffrey Chamberlain, Ph.D.

Vice President


My career has focused on muscle biology, diseases, and therapeutics. These activities span basic research on muscle gene structure and function to developing vectors for gene therapy of Duchenne muscular dystrophy (DMD). My graduate work involved cloning the muscle creatine kinase (MCK) gene and studying its regulation during development. This work (with Steve Hauschka) led to identification of the first muscle-specific enhancer element, which is now being used in multiple human gene therapy trials. As a post-doc with Tom Caskey I cloned mouse dystrophin while characterizing murine models for DMD. We also developed the method of multiplex PCR and applied it to several diseases, including prenatal diagnosis and carrier detection of DMD. Research in my own lab focused on structure/function studies of dystrophin, which led into studies of gene therapy. These efforts have centered around developing dystrophin expression cassettes and delivery vectors. We generated the first transgenic mouse models for DMD, which showed the feasibility of gene therapy, and pioneered development of mini- and micro-dystrophin genes. Much of our later work focused on vector systems and stems cells, including gutted adenoviral vectors, lentiviral vectors, and AAV. My lab was the first to show that AAV could be used for systemic gene delivery to muscle. This work led to the development of muscle-specific micro-dystrophin vectors capable of whole body treatment. Two biotech companies are using versions of our AAV/micro-dystrophin vectors in human gene therapy trials, and a third is testing our adenoviral vectors in COVID-19 and cancer vaccination trials.

2021

ASGCT Policy Summit

September 22-24, 2021

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