USA and United KingdomResearch and clinical trials
Gene therapy for SCID due to ADA deficiency: no deaths among 62 patients over a median 7.5 years
A study published in a journal and reviewed by independent scientists. Even so, a single study is not conclusive; it needs to be replicated.
Long-term results of stem cell gene therapy for severe combined immunodeficiency due to ADA deficiency have been published. In total, 33 patients were treated in the USA and 29 in the United Kingdom. All patients were alive at a median follow-up of 7.5 years. In 59 patients, the treatment took hold permanently, and enzyme therapy was no longer needed. Of these, 58 stopped immune support therapy, and no uncontrolled growth of blood cells was seen.
What does it mean for patients and families?
The results are strong, but this gene therapy was given within a study and is only for SCID due to ADA deficiency. For other types of SCID, options should be discussed with the immunology and transplant team.
Source
Scientific paper: Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency. The New England Journal of Medicine, 2025. DOI: 10.1056/NEJMoa2502754 · PubMed 41092330
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
Related disease: Severe combined immunodeficiency (SCID)
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.