Jim Wilson has 2 new promising gene therapy studies, but says investment in the cutting-edge field has ‘gotten worse’

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Wilson speaks about the Penn studies and the recent mixed results for Sarepta's muscular dystrophy drug.

Jim Wilson, director of the University of Pennsylvania's gene therapy program, led two new studies on the long-term effects of the cutting-edge treatments.gene-therapy scientist Jim Wilson has published two new studies supporting the promise of the cutting-edge field, finding evidence that the genetic treatments can be beneficial for years without raising the risk of cancer.

In one of the new studies, the Penn team confirmed that these genetic insertions occurred in lab monkeys, but in locations that were not expected to lead to cancer. “In monkeys and humans that have undergone gene therapy, we’ve never seen a formation of a tumor,” he said. Wilson and his colleagues measured the level of expression from the corrective genes administered to monkeys — that is, the level of beneficial proteins made from the “recipe” carried in those genes.

 

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