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18 May 2020

In memory of Jon A Wolff

Illustration for article: In memory of Jon A Wolff
It is with great sorrow that we share the sad news of the death of a great researcher, a pioneer of gene therapy and a fellow traveler of the AFM-Telethon. Jon A Wolff died on April 17, 2020 of esophageal cancer, the first signs of which he had detected during a visit to France 18 months earlier.

Jon was a rare person, whose intelligent mind was equalled only by his nobility of heart. He was a brilliant scientist, curious about everything, who combined his training as a pediatrician doctor with a solid education in molecular biology. He became a major player in medical research. He forged the first gene transfer tools, many of which are stirring current treatments. He has inspired many scientists all around the world.

He received his undergraduate education in Chemistry from Cornell University in New York State and his M.D. from the Johns Hopkins School of Medicine, followed by clinical training in Pediatrics and Medical Genetics at the University of San Diego, where he also conducted a postdoctoral fellowship at the Agouron Institute. In 1988, he became Assistant Professor and later on Professor of Pediatrics and Medical Genetics at the University of Wisconsin at Madison Medical School. He led its division of genetics and metabolism in Pediatrics for many years.

His scientific contribution was major. He published a multitude of diagnostic and therapeutic advances that became standards-of-care for many genetic diseases. Jon was instrumental during the great period of gene therapy in the 1980-90s, particularly in the development of DNA vectors and their applications to liver, brain and muscle therapies. He collaborated for several years with AFM and the French company Transgene on the gene therapy of Duchenne muscular dystrophy program that led in France to the world's first gene transfer clinical trial of a neuromuscular disease.

Some of his 80 patents are operated by the largest biotechnology companies, including Mirus Corp, which he founded in 1995, specialized in nanotechnology for the in vivo transfer of siRNA, and acquired in 2008 by the pharma giant Roche and then Arrowhead Research Corporation.

He founded and chaired the Genetic Support Foundation in 2012 and he served as advisor to the State of Wisconsin's Newborn Screening Program, as member of the Recombinant Advisory Committee of the U.S. National Institute of Health, as board member of the American Society of Gene Therapy, and as member of our scientific board.