She Was Born Without an Immune System. Gene Therapy Saved Her Life.

The article explains how gene therapy saved the life of a young girl, Cora Oakley, who was born with severe combined immunodeficiency (SCID). SCID is a rare genetic disorder that leaves babies without a functioning immune system. SCID is often referred to as "bubble boy disease," and it used to be considered a death sentence. Patients with this condition traditionally receive a bone-marrow transplant, but that carries serious risks, including graft vs host disease, long hospitalizations, infertility, and long-term organ damage.

The young child had ADA-SCID, which is one of the few variants of SCID that is targeted by a new form of a gene therapy trial from 2017. Instead of trying to replace her immune system, her doctors decided to collect her stem cells, insert a healthy ADA gene using a modified HIV virus, and return the corrected cells to her body. 

A study done recently that followed 62 babies found that every child was alive after eight years, and 95% had a fully restored immune system. The treatment for this condition is now much less dangerous for patients, and families are overall happier with this treatment. Although this treatment is costly for families.

This type of gene therapy saved Cora's life, as well as many other patients. She and many others can now live a healthy, normal life due to gene therapy. This article was refreshing to read to see good news occurring because of gene therapy.

References:

Bajaj, S. (2025, December 1). Gene therapy offer a cure for babies with the deadly ‘bubble boy disease’ - the new york times. https://www.nytimes.com/2025/11/27/well/bubble-boy-disease-gene-therapy.html

Wadbudhe, A. M., Meshram, R. J., & Tidke, S. C. (2023, October 26). Severe combined immunodeficiency (SCID) and its new treatment modalities. Cureus. https://pmc.ncbi.nlm.nih.gov/articles/PMC10676291/