Gene editing tools to protect and restore sight in inherited retinal diseases
Official title: Developing gene editing platforms for retinal degeneration.
Funded at Oregon State University · NIH-11384124 · Data updated 2026-11-12
A potential gene-editing therapy using prime editing and targeted lipid nanoparticles to treat inherited retinal diseases that can cause blindness.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Oregon State University NIH-funded |
| Lab location | 1 site (Corvallis, United States) |
| Project start | September 30, 2022 |
| Project end | June 30, 2027 |
| Funding year | 2026 |
| Award (this year) | $609,039 |
| NIH project number | 5R01EY033423-05 |
| Project ID | NIH-11384124 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals diagnosed with inherited retinal dystrophies caused by genetic mutations targetable by prime editing.
✕Less likely to help
Patients whose disease is not caused by the targetable mutations or who have advanced degeneration beyond recovery may not benefit.
What this research studies
This project aims to develop gene editing methods to fix genetic mutations causing retinal dystrophies, focusing on delivering editing tools directly to retinal cells. Researchers will explore prime editing capabilities and how to package these tools in lipid nanoparticles that reach the retina. The work includes optimizing delivery to retinal pigment epithelium and photoreceptors to correct disease-causing variants. If successful, the approach could lead to new, targeted treatments that preserve or restore vision for people with IRDs.
Why it matters
Potential benefit: If successful, patients with inherited retinal diseases could gain new options to protect or regain vision.
How similar studies have gone: Prime editing and targeted delivery have shown promise in other contexts, but this application to IRDs remains early-stage and exploratory.
Where this research is happening
-
Oregon State University
Corvallis, United States Active
Researchers
- Principal investigator: Sahay, Gaurav — Oregon State University
- Study coordinator: Sahay, Gaurav
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.