Improving nanoparticle delivery for gene editing in the retina
Optimizing lipid nanoparticles for retinal gene editing in the NHP
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 ID | NIH-11384237 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: People with inherited retinal dystrophies caused by an identifiable genetic mutation who might be candidates for future gene-editing therapies.
Not a fit: 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.
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 performed: 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
Portland, UNITED STATES
- 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
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.