Meet a PennMedicine Researcher


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James M. Wilson, MD, PhDJames M. Wilson, MD, PhD

James M. Wilson, MD, PhD, Director of the Gene Therapy Program, Interim Director of the Center for Orphan Disease Research and Therapy, and a Professor in the Department of Pathology and Laboratory Medicine, is the winner of the 2014 William Osler Patient Oriented Research Award. This award is granted to a member of the Perelman School of Medicine faculty for a body of work with an emphasis on clinical research.

During his 21 year career at Penn, Dr. Wilson revolutionized the preclinical use of gene transfer as a biological tool in basic and translational research, which rapidly advanced the progress of clinical gene therapy. His discovery of novel adeno-associated virus (AAV) serotypes and demonstration of the ability of gene transfer vectors to provide remarkably high transduction efficiency and stability of expression, with little to no immunogenicity, has changed the field of clinical gene therapy.

His most recent contribution is the discovery of an expanded family of AAVs from primates, including humans, and their development as gene therapy vectors. Of the AAV gene therapy trials submitted to the NIH’s Recombinant DNA Advisory Committee over the last 2 years, 75% of them involved vectors discovered by Dr. Wilson. Dr. Wilson is the epitome of a physician-scientist who has transformed clinical research and the treatment of many diseases.