Medicine: Giant Step for Gene Therapy

An experiment on a young girl opens a new era in the fight against hereditary diseases

She weighed around 35 lbs., came down with an occasional cold or ear infection, and appeared to be a healthy four-year-old. But a dark cloud hung . over her future. She suffered from ADA deficiency, the rare, incurable and deadly genetic disease that shuts down the immune system -- a disorder similar to the one that in 1984 finally claimed the life of David, the famous but unfortunate "bubble boy." Only the weekly injection of a newly developed drug seemed to stand between the little girl and the same fate.

Last week, on the 10th floor of the massive Clinical Center...

Want the full story?

Subscribe Now


Learn more about the benefits of being a TIME subscriber

If you are already a subscriber sign up — registration is free!