GlobalResearch and clinical trials
Gene therapy given to one eye in LHON: similar improvement seen in both eyes over 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.
A gene therapy in patients with LHON due to an MT-ND4 gene change was followed for 5 years. The treatment was given by injection into one eye, while a sham procedure was performed on the other eye. The long-term follow-up included 62 patients. Vision improved by similar amounts in both eyes compared with its worst level. In 66% of patients, there was a marked improvement in at least one eye.
What does it mean for patients and families?
Similar improvement in both eyes makes the effect of the treatment harder to interpret. Access in Türkiye depends on TİTCK (Turkish Medicines and Medical Devices Agency) and SGK (Social Security Institution) processes. Decisions should be made with an eye doctor.
Source
Scientific paper: Five-Year Outcomes of Lenadogene Nolparvovec Gene Therapy in Leber Hereditary Optic Neuropathy. JAMA Ophthalmology, 2025. DOI: 10.1001/jamaophthalmol.2024.5375 · PubMed 39699886
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: Leber hereditary optic neuropathy (LHON)
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.