Gene Therapy Advances To Better Treat 'Bubble Boy' Disease
The latest advance is not only encouraging news for patients with severe combined immunodeficiency. It's a test case for all those scientists working to develop better gene therapy techniques.
Sometimes rare diseases can let scientists pioneer bold new ideas. That has been the case with a condition that strikes fewer than 100 babies a year in the United States. These infants are born without a functioning immune system.
The disease is called severe combined immunodeficiency, or SCID. "It was made famous in the mid '70s when the 'Bubble Boy' was described in a documentary, and I think it captured the imagination of a lot of people," says Matthew Porteus, a pediatrician at Stanford University.
David Vetter was the boy who spent most of his short from infection. He died at age 12 in 1984.
