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CAMBRIDGE, Mass. — Gene therapy pioneer Dr. James Wilson is disappointed by the progress in his field — and expects current therapies and technologies to be soon surpassed by new approaches.

“In five years, when we look back on the way we’re executing on gene therapy now, we’re going to realize that things are going to be very different,” Wilson said at the STAT Summit on Thursday. “The way in which we’re going to treat Duchenne muscular dystrophy, potentially cure it, is not the way in which it’s being evaluated in the clinic now.”


Wilson, the director of the gene therapy program at the University of Pennsylvania’s medical school, knows a thing or two about disappointment. In 1999, Wilson ran an experiment that resulted in the death of Jesse Gelsinger, an 18-year-old with a mild form of a rare liver defect who became the first known patient to die in a clinical trial for gene therapy.

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