Immune Systems of ‘Bubble Babies’ Restored by Gene Therapy, UCLA Researchers Find
UCLA stem cell researchers have found that a gene therapy regimen can safely restore immune systems to children with so-called “bubble boy” disease, a life-threatening condition that if left untreated can be fatal within one to two years.
In the 11-year study, researchers were able to test two therapy regimens for 10 children with ADA-deficient severe combined immunodeficiency (SCID), which has come to be known as “bubble boy” disease because some of its victims have been forced to live in sterile environments. (more…)