Study: Gene therapy superior to half-matched transplant for 'bubble boy disease'

Children with SCID-X1 are born with a genetic defect that prevents them from developing a normal immune system. Because they are prone to life-threatening infections, infants with SCID-X1 must be kept in a sterile, protective bubble and require extensive treatment for survival beyond infancy.

http://www.eurekalert.org/pub_releases/2015-04/asoh-sgt040915.php