Once a ‘Refugee,’ a Gene Therapy Pioneer Finds a Renewed Calling as the Field Advances

February 18, 2019

(STAT News) – 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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