Pioneering ‘refugee’ from gene therapy’s darker days ‘kept the faith’

2-18-2019 jim wilson mini e x
James Wilson. Image credit: Ben Fidler

Gene therapy, now a pillar of biotechnology with the potential to cure deadly diseases, was once a cautionary tale of scientific brinkmanship.

At the turn of the century, the once-promising idea of replacing a faulty gene with a corrective copy took a tragic turn. A 1999 clinical trial resulted in the death of an 18-year-old patient, dashing the ambitions of researchers and beginning a years-long fallow period in which finding support — and funding — for gene therapy research was more difficult than ever.

“I refer to the group that continued to work at it as ‘refugees,’” said Dr. James Wilson, a physician and scientist at University of Pennsylvania who has studied gene therapy for more than three decades. “That’s how we would refer to one another.”

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Wilson, who led the 1999 trial, was among those who kept the faith.


He shared his work with that refugee network, and the field gradually turned a corner. In 2017, a company called Spark Therapeutics won the first-ever Food and Drug Administration approval for a gene therapy.

Over the past two decades, Wilson has been at once a proponent of gene therapy’s potential and a persistent voice of caution when it comes to the technology’s risks.


Read full, original post: Once a ‘refugee,’ a gene therapy pioneer finds a renewed calling as the field advances

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