Blocking NR2E3 to protect the eye's light-sensing cells
Targeting Nr2e3 to prevent photoreceptor degeneration
This project looks at whether turning off a gene called NR2E3 can protect the retina's photoreceptors in retinitis pigmentosa.
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 ID | NIH-11290321 on NIH RePORTER |
What this research studies
Researchers will use several mouse models of retinitis pigmentosa to see if removing the rod-specific gene NR2E3 can stop or slow photoreceptor loss. They will compare animals with NR2E3 removed during development to animals treated later with an AAV-delivered CRISPR-Cas9 system to knock out NR2E3, and will also compare the effects of increasing NR2E3. The team will measure molecular, cellular, physiological, and behavioral indicators of vision and cell survival across multiple genetic models of RP. These preclinical results could point to gene-independent ways to protect photoreceptors across many different RP mutations.
Who could benefit from this research
Good fit: People with retinitis pigmentosa or other inherited rod photoreceptor disorders would be the eventual candidates for therapies based on this work.
Not a fit: Patients with severe, end-stage retinal degeneration who have already lost most photoreceptors or people with non-RP retinal diseases are unlikely to benefit directly from this approach.
Why it matters
Potential benefit: If successful, this approach could lead to treatments that slow or prevent vision loss across many genetic forms of retinitis pigmentosa.
How similar studies have performed: AAV-CRISPR approaches have shown promise in animal retinas, but targeting NR2E3 as a broad, gene-independent protective strategy is relatively new.
Where this research is happening
Saint Louis, United States
- 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 below.