How special very-long-chain fats affect retinal health and macular degeneration
Elucidating the Role of Very-long-chain Polyunsaturated Fatty Acids in Retinal Health and Disease
This work looks at whether rare very-long-chain fats made in the eye help protect vision in people with Stargardt-type macular dystrophy and dry age-related macular degeneration.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Utah State Higher Education System--University of Utah NIH-funded |
| Lab location | 1 site (Salt Lake City, United States) |
| Project ID | NIH-11109459 on NIH RePORTER |
What this research studies
From a patient's point of view, this project focuses on unusual very-long-chain polyunsaturated fatty acids (VLC-PUFAs) that are made in the retina and may keep photoreceptor membranes flexible and working. Researchers use animal models (mice and zebrafish), genetically altered cells, and analyses of donated human retinas to track how VLC-PUFA levels and n-3/n-6 ratios change with ELOVL4 mutations and with dietary precursors. They measure retinal structure and function in animals lacking ELOVL4 and compare lipid profiles from human donor eyes and diet-related experiments. Findings are aimed at guiding whether dietary changes or targeting lipid pathways could help people with Stargardt-like disease or dry AMD.
Who could benefit from this research
Good fit: Ideal candidates would be people with Stargardt macular dystrophy (STGD3), those with atrophic (dry) age-related macular degeneration, or individuals willing to donate retinal tissue or take part in diet-related studies.
Not a fit: People with eye conditions not driven by photoreceptor membrane/lipid defects (for example primary glaucoma or diabetic retinopathy), or those seeking immediate approved therapies, are unlikely to gain direct benefit from this basic/translational research.
Why it matters
Potential benefit: Could point to dietary or molecular approaches that preserve photoreceptors and slow vision loss in Stargardt disease and dry AMD.
How similar studies have performed: Previous animal knockout experiments and analyses of human donor eyes show that VLC-PUFA loss and dietary precursors alter retinal lipid profiles and can cause photoreceptor dysfunction, but treatments based on this biology remain experimental.
Where this research is happening
Salt Lake City, United States
- Utah State Higher Education System--University of Utah — Salt Lake City, United States (Active)
Researchers
- Principal investigator: Bernstein, Paul Steven — Utah State Higher Education System--University of Utah
- Study coordinator: Bernstein, Paul Steven
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.