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NIH Committee Suggests Changes Following Fatal Gene Therapy Experiment

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The researcher, and his colleagues from the University of Pennsylvania, explained the complex set of events that led to Jesse's death. After the gene infusion, Gelsinger's temperature began to rise as expected, but then for unknown reasons his immune system went on a rampage, attacking his liver, his lungs, and his brain.

Wilson says autopsy findings suggest that perhaps Jesse had a viral infection at the time of treatment and that that, in combination with the cold virus, caused a pathological spike in inflammatory cells that lead to organ failure.

"We didn't promise we would succeed," says Wilson. However, he did promise to try again. "The story isn't over with Jesse; we still have more to do. Our hope is that we've started a dialogue. ... Is there any way we could redesign the vector to avoid this kind of problem?" asks Wilson.

The committee seemed sympathetic, and chairwoman Mickelson pointed out there is overwhelming evidence that gene therapy using a cold virus isn't unduly toxic. "If there were breaches in process, then I think that part of what happened [during the hearings] is recognition of that," says Mickelson.

Jesse Gelsinger's father Paul has sat through the hearings, trying to find meaning and consolation for the loss of his son. "I was a very discouraged man when I came here, but I am very much alive. My son has shown me how to live. That has sustained me through all of this," he tells WebMD.

The RAC has already gone on record saying it expects gene therapy researchers to be more forthcoming in disclosures about their studies.

 

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