Success Stories

The verdict on the pioneering children of gene therapy: so far, so good

To all outward appearances, Ashanthi ("Ashi") DeSilva is a normal, healthy 12-year-old who loves sports and would rather play basketball than do her seventh-grade homework. But Ashi holds a unique place in medical history: she is the first recipient of successful gene therapy.

Ashi was born with a rare, inherited disorder called ADA deficiency, the disease that claimed the life of the famed "bubble boy" in 1984. Because of a faulty gene, the T cells of her immune system were unable to produce an enzyme, ADA, necessary for their survival. As they died off, Ashi's immune system virtually shut down, leaving...

Want the full story?

Subscribe Now

Subscribe
Subscribe

Learn more about the benefits of being a TIME subscriber

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