Scientists from the US and the UK have taken an important step in treating a rare inherited disease — severe combined immunodeficiency caused by adenosine deaminase deficiency (ADA-SCID), better known as “bubble boy disease.” For the first time, a one-time gene therapy enabled 95% of children to restore full immune system function.
The study included 62 children who received their own stem cells modified with a working gene. In 59 patients, the immune system was fully restored. Nearly half were treated using frozen cells, which makes the method more convenient and accessible. The therapy proved safe and effective after 7.5 years of follow-up.
ADA-SCID was identified in the 1950s and 1960s, and its tragic symbol became David Vetter, a boy born in 1971 who spent his entire life until age 12 in a sterile “bubble” because he had no immune system at all. Today, half a century later, thanks to gene therapy, 95% of children with this diagnosis have a chance to restore their immune system and live a full life without constant fear.
