Creating gene editing tools for treating retinal degeneration.
Developing gene editing platforms for retinal degeneration.
['FUNDING_R01'] · OREGON STATE UNIVERSITY · NIH-10857288
This study is working on a new way to fix the genetic problems that cause vision loss in people with inherited retinal dystrophies by using a special gene editing tool and tiny particles to deliver it right to the eye, making treatments more effective for those affected.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | OREGON STATE UNIVERSITY (nih funded) |
| Locations | 1 site (CORVALLIS, UNITED STATES) |
| Trial ID | NIH-10857288 on ClinicalTrials.gov |
What this research studies
This research focuses on developing advanced gene editing techniques to address inherited retinal dystrophies (IRDs), which are genetic conditions leading to vision loss and blindness. The team aims to optimize a new genome editing tool called prime editing, which can correct a wide range of genetic mutations responsible for these conditions. By using lipid-based nanoparticles to deliver these gene editors directly to the eye, the research seeks to improve the effectiveness of treatment for patients with various genetic mutations. The approach involves isolating specific peptides to enhance the delivery of these treatments to the retinal cells that need them most.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with inherited retinal dystrophies caused by specific genetic mutations.
Not a fit: Patients with retinal degeneration not caused by genetic mutations or those with conditions unrelated to inherited retinal dystrophies may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option for patients suffering from inherited retinal dystrophies, potentially restoring vision.
How similar studies have performed: While gene editing is a rapidly evolving field, the specific application of prime editing for retinal diseases is still in the early stages, making this research a novel approach.
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.