Protecting cone vision in retinitis pigmentosa with targeted CRISPR therapy
Enhancing cone survival in retinitis pigmentosa through cell-specific therapeutic CRISPR editing of a roxadustat target
This project uses precise CRISPR gene editing to help cone photoreceptors survive in adults with retinitis pigmentosa.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Columbia University Health Sciences NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project ID | NIH-11124003 on NIH RePORTER |
What this research studies
You would be learning about a lab-led effort to reprogram metabolism in rod and cone cells so cones keep working after rods die in retinitis pigmentosa. The team plans cell-specific CRISPR edits that target the same pathway affected by the drug roxadustat and deliver these edits using viral vectors. Work includes lab and animal tests of cone survival and safety studies needed to support a future regulatory filing so human trials could begin. The ultimate aim is a one-time or limited treatment to slow vision loss across many different genetic causes of RP.
Who could benefit from this research
Good fit: Adults (over 21) with retinitis pigmentosa who still retain some cone vision and are willing to travel to a research center may be ideal candidates.
Not a fit: People with very advanced disease who have already lost most cone function, those with non-RP retinal diseases, or children under 21 are unlikely to benefit from this project.
Why it matters
Potential benefit: If successful, the approach could preserve central/daylight vision for many years in people with retinitis pigmentosa regardless of their specific gene mutation.
How similar studies have performed: While some gene-specific retinal therapies have helped certain patients, this metabolic/CRISPR strategy is novel and largely untested in humans so far.
Where this research is happening
New York, United States
- Columbia University Health Sciences — New York, United States (Active)
Researchers
- Principal investigator: Tsang, Stephen H — Columbia University Health Sciences
- Study coordinator: Tsang, Stephen H
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.