Preventing vision loss by targeting a specific gene in the retina
Official title: Targeting Nr2e3 to prevent photoreceptor degeneration
Funded at Washington University · NIH-11044998 · Data updated 2026-11-12
This study is exploring a new way to help people with retinitis pigmentosa by looking at how changing a specific gene can stop vision loss, using mice to find the best method for protecting eye cells.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Washington University NIH-funded |
| Lab location | 1 site (Saint Louis, United States) |
| Project start | March 1, 2023 |
| Project end | February 28, 2028 |
| Funding year | 2025 |
| Award (this year) | $502,086 |
| NIH project number | 5R01EY033810-03 |
| Project ID | NIH-11044998 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with retinitis pigmentosa or those at risk of photoreceptor degeneration.
✕Less likely to help
Patients with retinal conditions unrelated to photoreceptor degeneration may not benefit from this research.
What this research studies
This research investigates a potential treatment for retinitis pigmentosa, a leading cause of blindness, by targeting the Nr2e3 gene to prevent photoreceptor degeneration. The study will utilize various mouse models to assess the effectiveness of knocking out this gene using advanced techniques like CRISPR-Cas9 and adeno-associated virus delivery. By examining both developmental and acute knockout strategies, the research aims to determine the best approach to protect retinal cells from degeneration. Patients may benefit from insights gained that could lead to new therapies for vision preservation.
Why it matters
Potential benefit: If successful, this research could lead to innovative treatments that prevent vision loss in patients with retinitis pigmentosa.
How similar studies have gone: Previous research has shown promise in targeting gene-independent therapies for retinal diseases, suggesting potential success for this approach.
Where this research is happening
-
Washington University
Saint Louis, United States Active
Researchers
- Principal investigator: Corbo, Joseph — Washington University
- Study coordinator: Corbo, Joseph
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.