For many, hearing the term “bubble boy” conjures up images of John Travolta in “The Boy in the Plastic Bubble” or Jake Gyllenhaal in the 2001 box office flop “Bubble Boy.” Whatever your preferred point of reference, the idea of a child spending his life in a literal “bubble” of plastic is a fate all too real for some families in the last five or so decades.
ADA-deficient severe combined immunodeficiency, or SCID, is a disease that leaves a child’s immune system unable to fight off germs or bacteria. Most children diagnosed with the disease die within the first or second year of life. But now, a breakthrough gene therapy offers new hope to families of children with this rare disease, according to today’s announcement by the UCLA Eli & Edith Broad Center of Regenerative Medicine and Stem Cell Research.
Doctor Denise Cabonaro-Sarriciono helped to develop the new treatment. She joins Larry Mantle to talk about it.
Dr. Denise Carbonaro-Sarricino, Project Scientist at the UCLA Broad Stem Cell Research Center and member of the team that developed the new treatment.