Protecting cone vision in retinitis pigmentosa with targeted CRISPR therapy
Official title: Enhancing cone survival in retinitis pigmentosa through cell-specific therapeutic CRISPR editing of a roxadustat target
Funded at Columbia University Health Sciences · NIH-11124003 · Data updated 2026-11-12
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 start | June 1, 2022 |
| Project end | May 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $450,345 |
| NIH project number | 5R01EY033770-04 |
| Project ID | NIH-11124003 on NIH RePORTER |
Who could benefit from this research
✓Could help
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.
✕Less likely to help
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.
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.
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 gone: 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
-
Columbia University Health Sciences
New York, United States Active
Researchers
- Principal investigator: Tsang, Stephen H — Columbia University Health Sciences
- Study coordinator: Tsang, Stephen H
Related research
Other NIH-funded projects on the same topics.
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Inflammation in MERTK-related retinitis pigmentosa
This project aims to understand whether losing the MERTK gene causes retinal inflammation that leads to vision loss in people with MERTK-related retinitis pigmentosa.
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Enhancing cone survival in RP through targeted cell-specific CRISPR therapy
A patient-friendly look at a new approach to protect cone cells in retinitis pigmentosa using precise gene editing to support vision.
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Oral, once-daily MC16 medicine for retinitis pigmentosa
A gene-mutation-agnostic pill (MC16) is being studied to help people with retinitis pigmentosa by addressing a common cellular problem in the eye.
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Role of IPM Proteoglycans in Retinal Health and Inherited Blindness
This grant studies how specific retinal extracellular matrix proteins (IMPG1 and IMPG2) affect eye health and contribute to retinitis pigmentosa, with potential implications for people who have or are at risk for inherited blindness.
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NAC Attack: a late-stage trial for preserving vision in RP patients
A large, multi-site study is testing whether taking a daily NAC pill can help people with retinitis pigmentosa keep more of their vision.
-
Non-invasive biomarker for mitochondria health in retinitis pigmentosa
Aims to test a new, non-invasive OCT-based biomarker to explain and predict how mitochondria function affects cone cells in people with retinitis pigmentosa.
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.