Improving nanoparticle delivery for gene editing in the retina
Official title: Optimizing lipid nanoparticles for retinal gene editing in the NHP
Funded at Legacy Emanuel Hospital and Health Center · NIH-11384237 · Data updated 2026-11-12
This project develops lipid nanoparticle delivery of temporary gene-editing tools to help people with inherited retinal dystrophies that cause vision loss.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Legacy Emanuel Hospital and Health Center NIH-funded |
| Lab location | 1 site (Portland, UNITED STATES) |
| Project start | July 1, 2024 |
| Project end | April 30, 2029 |
| Funding year | 2026 |
| Award (this year) | $689,274 |
| NIH project number | 5R01EY035258-04 |
| Project ID | NIH-11384237 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with inherited retinal dystrophies caused by an identifiable genetic mutation who might be candidates for future gene-editing therapies.
✕Less likely to help
People with vision loss from non-genetic causes, very advanced retinal degeneration with few remaining target cells, or diseases driven by genes not addressed by this approach are unlikely to benefit.
What this research studies
Researchers are designing and optimizing lipid nanoparticles (LNPs) that can carry mRNA-based gene-editing tools into retinal cells. They will test delivery, dosing, safety, and editing efficiency in non-human primates to better mimic the human eye. The goal is to create a transient, controllable delivery method that avoids long-term viral expression. Data from these preclinical tests will guide whether this approach could move toward human trials.
Why it matters
Potential benefit: If successful, this could enable safer and more precise gene-editing treatments to slow or restore vision loss from many inherited retinal diseases.
How similar studies have gone: An AAV-based retinal CRISPR therapy entered early clinical trials (EDIT-101) but is paused; lipid nanoparticle mRNA delivery has shown success in vaccines and liver therapies but is newer and experimental for the retina.
Where this research is happening
-
Legacy Emanuel Hospital and Health Center
Portland, United States Active
Researchers
- Principal investigator: Ryals, Renee Christine — Legacy Emanuel Hospital and Health Center
- Study coordinator: Ryals, Renee Christine
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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.