Lasting Cure Found for ADA-SCID Through Gene Therapy

 Lasting Cure Found for ADA-SCID Through Gene Therapy

    ADA deficiency inhibits the development and function of immune cells (due to a genetic mutation), usually combined with SCID, in which people lack all immune protection from bacteria, viruses, and fungi (Adenosine Deaminase Deficiency: MedlinePlus Genetics, 2013). A child named Eliana was born with this disorder since she was a baby and lived inside a "bubble" ever since, until researchers using experimental gene therapy cured the child's immune system when she was 10 months old, and she no longer lives in the bubble!

In the new gene therapy, the child's own blood stem cells were collected and treated with a modified virus that implanted a healthy copy of the ADA gene. These stem cells are now infused back into the child, where the healthy cells start producing immune cells to fight infections. For the immune system to reach normal levels, it takes about 6 months to a year.

This long-term research (2012-2019) included 62 patients, and only 59 were successfully treated with no complications! The gene therapy has not yet been FDA-approved, but researchers are preparing to apply!

A photo of Eliana Nachem

This is really important research because of the lives that could be saved with this gene therapy. Gene therapy is a great method because it targets the root of the problem instead of just treating symptoms. Gene therapy also gives hope to parents and families of those affected by ADA-SCID, just like Eliana's family. 

References

Adenosine deaminase deficiency: MedlinePlus Genetics. (2013, July 1). Medlineplus.gov. https://medlineplus.gov/genetics/condition/adenosine-deaminase-deficiency/

HealthDay. (2025). Gene Therapy Provides Lasting Cure For “Bubble Boy” Children. US News & World Report; U.S. News & World Report. https://www.usnews.com/news/health-news/articles/2025-10-16/gene-therapy-provides-lasting-cure-for-bubble-boy-children