Gene breakthrough after sad setback

The vector in that study triggered a fatal immune system overreaction. The fallout set off years of government investigations and efforts to improve human-subject protections.

"That tragic event forced me to reevaluate where we were and where the field was," Wilson said. "For this field to succeed, we had to go back to basics. I do believe this is a positive legacy to that."

Wilson publicly apologized and accepted responsibility for the death - almost a decade later.

While his remorse has been dismissed by critics such as Gelsinger's father, Paul, there is no doubt his research has dramatically advanced the science.

Since 2000, he and his team have overcome some of the biggest technological obstacles to harnessing altered viruses to infect cells with helpful, rather than harmful, DNA. They discovered 120 new adeno-associated viruses (AAV) in monkeys and humans - only six were previously known - and created an array of gene transporters. Through animal testing, they identified vectors with an affinity for certain tissues, enabling gene therapy to target organs such as the liver, eyes, and muscle.

Penn Vector Core, a university facililty that Wilson oversees, now provides viral and non-viral vectors - at cost - to academic researchers around the world. Through an affiliated company founded by Penn, ReGenX, biotech corporations can also access novel AAV vectors.

If companies that use Penn's technology make money with the resulting therapy, Penn - and in some cases, Wilson - stands to profit.

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Gene breakthrough after sad setback

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