Understanding how faulty myocilin protein harms eye cells in glaucoma
Molecular determinants of cytotoxicity due to mutant myocilin misfolding
['FUNDING_R01'] · GEORGIA INSTITUTE OF TECHNOLOGY · NIH-11178610
This project aims to discover why a specific faulty protein called myocilin damages eye cells, which could lead to new treatments for glaucoma.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | GEORGIA INSTITUTE OF TECHNOLOGY (nih funded) |
| Locations | 1 site (ATLANTA, UNITED STATES) |
| Trial ID | NIH-11178610 on ClinicalTrials.gov |
What this research studies
Glaucoma can lead to blindness, and current treatments don't fix the root cause, especially when it's linked to a faulty protein called myocilin. This protein, when mutated, doesn't work correctly and builds up inside eye cells, causing them to die. This cell damage leads to increased pressure inside the eye, which then harms the optic nerve and causes vision loss. Our goal is to uncover the exact ways this faulty protein harms eye cells, using a special yeast model to help us understand these processes.
Who could benefit from this research
Good fit: Patients with early-onset or inherited forms of glaucoma linked to myocilin mutations might eventually benefit from therapies developed from this research.
Not a fit: Patients whose glaucoma is not related to myocilin mutations or who have other forms of the disease may not directly benefit from this specific line of research.
Why it matters
Potential benefit: If successful, this work could reveal new targets for medications that directly address the underlying cause of glaucoma, potentially preventing vision loss.
How similar studies have performed: This research aims to establish the molecular pathways of cytotoxicity, which are not yet fully understood, making this a foundational and novel approach to developing disease-modifying therapies.
Where this research is happening
ATLANTA, UNITED STATES
- GEORGIA INSTITUTE OF TECHNOLOGY — ATLANTA, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: LIEBERMAN, RAQUEL L — GEORGIA INSTITUTE OF TECHNOLOGY
- Study coordinator: LIEBERMAN, RAQUEL L
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Conditions: Alzheimer disease dementia, Alzheimer syndrome, Alzheimer's Disease