Apr 21, 2002 3 min read

PHILADELPHIA (AP) – The director of a gene-therapy institute at The University of Pennsylvania is stepping down, three years after the death of a teen-age research subject led to questions about the safety of such research on humans. Dr. James Wilson, who came under fire after the 1999 death of 18-year-old Jesse Gelsinger, will continue to teach and conduct research at Penn after leaving his post July 1.

Meanwhile, the institute will broaden its focus because of the limited success that gene therapy has shown so far, said Penn’s medical school dean.

“The committee has recommended that the Institute broaden its scientific focus to include cell-based therapies, as well as stem cell biology and molecular virology,” Dean Arthur H. Rubenstein told The Philadelphia Inquirer.

“The promise in 1992 was like it would be a fantastic and wonderful thing for patients,” Rubenstein said. “…(I)t has proved to be extremely challenging, not just at Penn, but all over the world.”

Wilson came to the university in 1993 amid great hope that his gene-therapy research would someday cure a variety of diseases. But federal officials reviewing Gelsinger’s death found numerous policy violations and banned Wilson from further human trials involving gene therapy.

Gelsinger, who had a mild form of an inherited liver disease, came to Penn after learning that Wilson was working on the disease, called ornithine transcarbamylase (OTC) deficiency.

In the experiment, a genetically engineered cold virus was used to send copies of corrective genes into the body to treat or fix the disorder. But Gelsinger’s body launched an immune system response to the therapy that killed him.

“This move has been a long time coming and comes way too late for Jesse,” lawyer Alan Milstein, who represents Gelsinger’s family’s, said of Wilson’s resignation.

Wilson, who oversaw 100 people at the institute, still hopes to combat illnesses such as cystic fibrosis through gene therapy.

“I look forward to the opportunity to dedicate more of my time to teaching and research in the development of novel gene-delivery vehicles and their application in the treatment of diabetes, cystic fibrosis, and infectious diseases,” he said in a statement released Friday.

Wilson, a father of four, has both a medical degree and a doctorate in biological chemistry. He also became an entrepreneur, in 1992 forming a biotechnology company called Genovo Inc. that he hoped would market the gene therapy work he did in the laboratory. Wilson made $13.5 million when the company was sold to Targeted Genetics Corp. of Seattle eight years later.