User:Jon A Wolff

Jon A. Wolff, M.D. is a co-founder of Mirus, Chief Scientific Officer and a member of the Board of Directors. Dr. Wolff received his B.A. Degree in Chemistry at Cornell University and earned his M.D. at The Johns Hopkins University, School of Medicine. He completed his medical residency and a fellowship in genetics and metabolism at the University of California, San Diego, after which he served as an Assistant Professor for three years. Since joining the University of Wisconsin – Madison in 1988 as an Assistant Professor, he was appointed Director of the Biochemical Genetics Program at the Waisman Center in 1989, advanced to Professor in the departments of Pediatrics and Medical Genetics, and promoted to Director of the entire Clinical and Medical Genetics division in 2001.

Board certified in Pediatrics and Biochemical, Clinical, and Molecular Genetics, Dr. Wolff is an acknowledged leader in the field of gene therapy. Dr. Wolff was a member of the National Institute of Health's Recombinant DNA Advisory Committee from 1997-2000, responsible for oversight of human gene therapy trials. He is an associate editor for Molecular Therapy and is on the editorial board for the journals Human Gene Therapy, Gene Therapy, and Cancer Gene Therapy. He was recognized by the Harvard Medical Newsletter as one of the Top Ten Medical Advances of 1991 for his research in gene transfer. Recently, Dr. Wolff was elected to serve on the board of the American Society for Gene Therapy.

The aim of Dr. Jon Wolff’s current research is to develop and refine the ability to directly deliver plasmid DNA into muscle, liver and other tissues through further development of the basic scientific knowledge of how plasmid DNA traverses cell membranes, enters nuclei and attains stable expression. Dr Wolff has grants to develop and evaluate the delivery of “naked DNA” to muscle cells via an intravascular route and to identify peptides that prolong the circulation of gene vectors in the blood and enable targeting to specific tissues.

Dr. Wolff's data show that plasmid DNA can be delivered via a blood vessel into more than 10% of the muscle cells throughout the leg of a rat. This percentage of transfected muscle cells approaches the critical minimum percentage necessary to be curative in children with Duchenne muscular dystrophy. With this approach, multiple administrations should be possible, ensuring that a sufficient number of cells would express functional dystrophin, the missing protein in people with Duchenne muscular dystrophy.

Dr. Wolff’s studies also indicate that this approach should lead to stable expression of the gene. Work is now underway to extend this approach to larger animals. If successful in larger animals, a human clinical trial in patients with Duchenne muscular dystrophy could begin in the near future. In addition, work is underway to extend the intravascular gene delivery method to other organs, with a current emphasis on promoting stable gene expression in liver.