Beneath all the bad news about viruses this week lies a good virus: the one that underlies gene therapy for X-linked severe combined immunodeficiency (SCID-X1).
Altered viruses are the vehicles that transfer healthy human genes into the cells of people in whom the genes aren’t working, providing a slew of new “forever fixes.” Nearly 100 gene transfer protocols are now in late-stage clinical trials.
The long-anticipated research report in the New England Journal of Medicine sets gene therapy back on track, after a previous clinical trial treated the disease but triggered leukemia. The ongoing saga is also a terrific example of how scientific inquiry catalyzes medical progress.
SCID-X1 became widely known as “bubble boy disease” in 1976, when John Travolta played David Vetter in the film “The Boy In The Plastic Bubble.” It was a year after he rocketed to fame as sweathog Vinnie Barbarino on the TV program “Welcome Back Kotter.”
Read full, original article: SCID-X1 Gene Therapy, Take 2
