ChinaResearch and clinical trials
Single-dose gene therapy into the eye found safe in 12 children with X-linked retinoschisis
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 China, 12 patients aged 5 to 18 received gene therapy in one eye. Over one year, no serious side effects or eye inflammation were seen. One patient developed a hole in the macula (the central part of the retina). Vision in treated eyes improved by an average of 10.8 letters. In the other eye, the improvement was 2.4 letters. In all patients, the splitting in the macula closed.
What does it mean for patients and families?
This is a small, early-stage study. The treatment is not yet approved. Larger studies are needed.
Source
PubMed (New England Journal of Medicine): Subretinal Gene Therapy for X-Linked Retinoschisis.
Scientific paper: Subretinal Gene Therapy for X-Linked Retinoschisis. The New England Journal of Medicine, 2026. DOI: 10.1056/NEJMoa2515953 · PubMed 42269152
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: X-linked retinoschisis (XLRS)
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.