How repairing DNA through gene therapy overcame high-profile failures

Gene therapy pioneer Richard Jude Samulski remembers when he avoided the words “gene therapy.” In the mid-2000s, he told people he worked on “biological nanoparticles,” even attempting to trademark the term. “We felt that was the disguise we were going to have to wear to go forward,” recalls Samulski, a professor of pharmacology at the University of North Carolina at Chapel Hill.

The death of a teenager in a gene therapy clinical trial in 1999 and cases of leukemia in a trial soon after almost extinguished the field, which seeks to treat diseases at their roots by…