Gene‑editing approaches to treat inherited retinal degeneration
Developing gene editing platforms for retinal degeneration.
This project will try prime editing delivered with lipid nanoparticles to fix genetic mutations that cause inherited retinal dystrophies and help people with genetic vision loss.
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 ID | NIH-11115864 on NIH RePORTER |
What this research studies
Inherited retinal dystrophies are caused by hundreds of different gene changes that can lead to progressive vision loss and blindness. The team plans to use prime editing, a precise form of gene editing, packaged as mRNA inside lipid nanoparticles (LNPs) to make targeted DNA corrections in retinal cells. They will use phage display to find peptides that help LNPs reach photoreceptors as well as the retinal pigment epithelium, and test delivery and editing efficiency in relevant models. The work aims to optimize a nonviral, modular delivery system that could one day enable treatments for many different genetic retinal diseases.
Who could benefit from this research
Good fit: People with genetically confirmed inherited retinal dystrophies or other inherited retinal mutations that cause progressive vision loss would be the most likely candidates for related future trials.
Not a fit: Patients whose vision loss is due to non‑genetic causes or who have lost most photoreceptors already may not benefit from this gene‑correction approach.
Why it matters
Potential benefit: If successful, this could enable precise, potentially one‑time genetic fixes for many causes of inherited retinal degeneration and might preserve or restore vision.
How similar studies have performed: There is one FDA‑approved viral gene therapy for a specific retinal disease and LNPs have shown delivery to the retinal pigment epithelium, but delivering prime editors to photoreceptors in vivo is largely novel and unproven.
Where this research is happening
Corvallis, United States
- 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 below.