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James M. Wilson, MD, PhD

  • Director, Gene Therapy Program
  • Rose H. Weiss Professor and Director, Orphan Disease Center
  • Professor of Medicine and Pediatrics, Department of Medicine

“We are at the beginning of a revolution in the treatment of patients with rare diseases. The commitment of the staff and scientists at GTP to the development of treatments for rare disease patients inspires me every day I come into work."

Jim’s commitment to patients with rare diseases stemmed from his days as a graduate student and medical student at the University of Michigan between 1977 and 1984. He was discouraged by the lack of available treatments for rare disease patients but optimistic that science could yield solutions with his initial focus being gene therapy. He completed his training in Internal Medicine at the Massachusetts General Hospital followed by a postdoctoral fellowship at the Whitehead Institute where he began his work in gene therapy.

After launching his faculty career in the Howard Hughes Medical Institute the University of Michigan, he moved to the University of Pennsylvania in 1993. Jim’s laboratory has made seminal contributions to the technology of gene transfer and has paved the way for translation of these technologies into the clinic. He has published over 550 papers and is named on over 110 patents. Jim is a Trustee at Albion College and is the founder and President of a 501(c)3 called Health Through Fitness in Orphan Diseases.

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