USAResearch and clinical trials
Stem cell gene therapy for cystinosis: first results published in 6 adults
A study published in a journal and reviewed by independent scientists. Even so, a single study is not conclusive; it needs to be replicated.
In an early-stage study in the USA, 6 adults received gene therapy using their own stem cells. Patients were followed for 29 to 63 months. In five patients, cystine in blood cells decreased. Most side effects were mild or moderate and were related to the bone marrow preparation treatment.
What does it mean for patients and families?
This is a very small, early study. The treatment is not approved. Current drug treatment should not be stopped without a doctor's advice.
Source
PubMed (New England Journal of Medicine): Hematopoietic Stem-Cell Gene Therapy for Cystinosis.
Scientific paper: Hematopoietic Stem-Cell Gene Therapy for Cystinosis. The New England Journal of Medicine, 2026. DOI: 10.1056/NEJMoa2506431 · PubMed 41707137
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: Nephropathic cystinosis
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.