Gene Therapy Provides Lasting Cure For ‘Bubble Boy’ Children

Key Takeaways

An experimental genetic therapy can cure most cases of the ‘Bubble Boy’ disease, ADA-SCIDIn the rare disorder, children are born without a functioning immune system due to genetic mutationsThe therapy replaces broken genes with healthy copies, fully restoring their immune function within a year

THURSDAY, Oct. 16, 2025 (HealthDay News) — Eliana Nachem is taking a brave step once unthinkable for the 11-year-old girl — she’s starting sixth grade, with dreams of becoming an artist.

As a baby, Eliana lived in complete medical isolation, after her 2014 diagnosis at 3 months of age with ADA-SCID, more popularly known as “Bubble Boy” disease.

Kids with the rare disorder are born with a broken immune system caused by adenosine deaminase (ADA) deficiency, in which a genetic mutatio