Optineurin problems and nerve damage in normal-tension glaucoma

Optineurin dysfunction induces neurodegeneration in normal tension glaucoma by a novel molecular mechanism

NIH-funded research Stanford University · NIH-11259513

Researchers are using a new mouse model to discover how changes in the optineurin gene cause retinal nerve cell loss in people with normal-tension glaucoma and related nerve diseases.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionStanford University NIH-funded
Lab location1 site (Stanford, United States)
Project IDNIH-11259513 on NIH RePORTER

What this research studies

This project uses a newly developed mouse model that mimics the optic nerve and retinal ganglion cell loss seen in normal-tension glaucoma by truncating the optineurin (OPTN) gene in those eye cells. The team will identify and study proteins that interact with OPTN in retinal ganglion cells and along the optic nerve to map the steps that lead to cell death and axon degeneration. They will characterize the molecular pathways in damaged nerves to find points that could be protected or fixed. Because OPTN mutations also occur in some forms of ALS, findings may reveal common targets relevant to both eye and motor neuron degeneration.

Who could benefit from this research

Good fit: People with normal-tension glaucoma—especially those with known OPTN genetic variants—or patients interested in research on genetic causes of optic nerve degeneration would be most relevant to follow this work.

Not a fit: Patients seeking immediate treatment benefit or those with high-pressure glaucoma unrelated to OPTN changes are unlikely to gain direct clinical benefit from this basic-science grant.

Why it matters

Potential benefit: If successful, the work could point to molecular targets for treatments that slow or prevent vision loss in normal-tension glaucoma and possibly inform therapies for related neurodegenerative diseases.

How similar studies have performed: Previous studies have linked OPTN mutations to NTG and ALS and the investigators have created a rapid NTG mouse model, but translating these mechanistic findings into effective patient therapies remains unproven.

Where this research is happening

Stanford, United States

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Conditions Amyotrophic Lateral Sclerosis Motor Neuron Disease
Last reviewed 2026-06-09 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.