--- title: Gene-Therapy Trial Shows Promise Fighting 'Bubble Boy' Syndrome description: A new gene-therapy treatment is showing promise in treating a rare congenital condition that involves extreme immune-system deficiencies canonical: https://time.com/3483397/gene-therapy-trial-shows-promise-fighting-bubble-boy-syndrome/ author: Elizabeth Barber article:opinion: false article:content_tier: free article:published_time: 2014-10-09T08:08:08.000Z article:modified_time: 2026-02-21T10:40:30.429Z article:section: Health og:title: Gene-Therapy Hope for 'Bubble Boy' Syndrome og:description: A new gene-therapy treatment is showing promise in treating a rare congenital condition that involves extreme immune-system deficiencies og:url: https://time.com/3483397/gene-therapy-trial-shows-promise-fighting-bubble-boy-syndrome/ og:site_name: TIME og:image: https://static.time.com/v3/assets/bltea6093859af6183b/bltde093bcdab65f2d0/6988d9b9bc6cfc69913e2a3c/time-logo.jpg?branch=production og:image:width: 1200 og:image:height: 675 og:image:alt: TIME og:type: article twitter:card: summary_large_image twitter:title: Gene-Therapy Hope for 'Bubble Boy' Syndrome twitter:description: A new gene-therapy treatment is showing promise in treating a rare congenital condition that involves extreme immune-system deficiencies twitter:image: https://static.time.com/v3/assets/bltea6093859af6183b/bltde093bcdab65f2d0/6988d9b9bc6cfc69913e2a3c/time-logo.jpg?branch=production --- # Gene-Therapy Trial Shows Promise Fighting 'Bubble Boy' Syndrome by [Elizabeth Barber](https://time.com/author/elizabeth-barber/) ## Elizabeth Barber Oct 9, 2014 8:08 AM UTC by [Elizabeth Barber](https://time.com/author/elizabeth-barber/) ## Elizabeth Barber Oct 9, 2014 8:08 AM UTC A new gene-therapy treatment is showing promise in treating a rare and severe congenital condition that involves extreme immune-system deficiencies. “Bubble boy” syndrome, an X-linked condition, takes its name from a famous case in which an affected boy, vulnerable to infection, lived inside a plastic bubble that protected him from the world’s germs. Outside of such [sterile environments](http://online.wsj.com/articles/gene-therapy-trial-for-bubble-boy-syndrome-shows-promise-1412802002), babies with the syndrome seldom live longer than a year, the _Wall Street Journal_ reports. The condition has for decades bested medical researchers, despite occasional bouts of optimism — hope for one previous gene-therapy treatment was felled when some recipients developed leukemia. Gene-therapy treatment works, essentially, by replacing unperforming genes with functional ones. Dysfunctional cells are removed from the child’s immune system and exposed to a genetically engineered virus that can reprogram the cells to function properly, [explains](http://www.reuters.com/article/2014/10/08/us-gene-therapy-bubble-boy-idUSKCN0HX2B020141008) Reuters. Those cells are then reinserted back into the patient. In the earlier treatment, the virus to which the cells were exposed apparently activated a part of their genetic code that leads to leukemia, Reuters says. But initial [results](http://www.nejm.org/doi/full/10.1056/NEJMoa1404588) reported in the _New England Journal of Medicine_ show that none of the nine babies from the U.S. and Europe who received the latest treatment are exhibiting any signs of cancer. Of the nine infant participants in the research — who were between 4 and 10½ months old when they began receiving the therapy — eight were still alive 16 to 43 months later, without living in a protective “bubble.” (The ninth child died four months after treatment began from an earlier infection he had been fighting.) Out of the eight boys still living, the treatment upped blood T-cell levels, rebuilding the immune system, of seven. In the case of the eighth child, the treatment did not rebuild his immune system, but a successful stem-cell transplant has kept him in improved health, Reuters reports. ```json [{"@context":"https://schema.org","@type":"NewsArticle","@id":"https://time.com/3483397/gene-therapy-trial-shows-promise-fighting-bubble-boy-syndrome/","mainEntityOfPage":{"@type":"WebPage","@id":"https://time.com/3483397/gene-therapy-trial-shows-promise-fighting-bubble-boy-syndrome/"},"headline":"Gene-Therapy Trial Shows Promise Fighting 'Bubble Boy' Syndrome","datePublished":"2014-10-09T08:08:08.000Z","dateModified":"2026-02-21T10:40:30.429Z","description":"The immune system-related disease affects about 1 in 100,000 babies each year","url":"https://time.com/3483397/gene-therapy-trial-shows-promise-fighting-bubble-boy-syndrome/","keywords":["Genetics","immune system"],"thumbnailUrl":"","author":[{"@type":"Person","name":"Elizabeth Barber","jobTitle":null,"url":"https://time.com/author/elizabeth-barber/"}],"articleSection":"Health","image":[],"publisher":{"@type":"Organization","name":"Time","url":"https://time.com/","logo":{"@type":"ImageObject","url":"https://time.com/images/logo.png","width":528,"height":156},"foundingDate":"March 3, 1923","sameAs":["https://www.facebook.com/time","https://www.instagram.com/time/?hl=en","https://twitter.com/time","https://www.pinterest.com/timemagazine"]}},{"@context":"https://schema.org","@type":"BreadcrumbList","itemListElement":[{"@type":"ListItem","position":1,"item":{"@id":"/section/health/","name":"Health"}},{"@type":"ListItem","position":2,"item":{"@id":"/tag/genetics/","name":"Genetics"}},{"@type":"ListItem","position":3,"item":{"@id":"https://time.com/3483397/gene-therapy-trial-shows-promise-fighting-bubble-boy-syndrome/","name":"Gene-Therapy Trial Shows Promise Fighting 'Bubble Boy' Syndrome"}}]}] ```