New treatments for inherited optic atrophy using a primate model
Advancing novel therapies for optic neuropathy with a nonhuman primate model
This project uses a rhesus macaque model of OPA1-related inherited optic atrophy to speed development of better treatments for people with vision loss from this condition.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California at Davis NIH-funded |
| Lab location | 1 site (Davis, United States) |
| Project ID | NIH-11304502 on NIH RePORTER |
What this research studies
Researchers are characterizing rhesus macaques that carry the same OPA1 mutation seen in people with autosomal dominant optic atrophy and will follow how the optic nerve and retinal ganglion cells change over time. They will use advanced eye imaging, structural and functional vision tests, and comparisons to unaffected animals to define disease onset and progression over five years. The team will validate this primate model so it better predicts how therapies might work in humans and use it to refine candidate treatments that target mitochondrial dysfunction in retinal cells. Results are intended to make preclinical testing more relevant before moving potential therapies into human trials.
Who could benefit from this research
Good fit: People with autosomal dominant optic atrophy (confirmed or suspected OPA1 mutations) or those with inherited optic neuropathies interested in future trials or contributing samples would be most relevant.
Not a fit: Patients whose vision loss is due to unrelated causes (for example advanced glaucoma, vascular or traumatic optic neuropathy) may not receive direct benefit from this OPA1-focused project.
Why it matters
Potential benefit: If successful, this work could accelerate the development of therapies that preserve or restore vision for people with OPA1-related inherited optic atrophy.
How similar studies have performed: Animal models have informed other vision therapies, but well-characterized nonhuman primate models of OPA1-related optic atrophy are novel and not yet widely validated.
Where this research is happening
Davis, United States
- University of California at Davis — Davis, United States (Active)
Researchers
- Principal investigator: Thomasy, Sara Michelle — University of California at Davis
- Study coordinator: Thomasy, Sara Michelle
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.