Enhancing cone survival in RP through targeted cell-specific 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-11420299 · Data updated 2026-11-12
A patient-friendly look at a new approach to protect cone cells in retinitis pigmentosa using precise gene editing to support vision.
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 | 2026 |
| Award (this year) | $529,818 |
| NIH project number | 5R01EY033770-05 |
| Project ID | NIH-11420299 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with retinitis pigmentosa or related inherited retinal dystrophies who are potential candidates for future cone-protective therapies.
✕Less likely to help
People without RP or with non-metabolic causes of photoreceptor loss may not benefit.
What this research studies
This project aims to help people with retinitis pigmentosa by using a CRISPR-based approach to reprogram the metabolism of the retina so cones survive longer. Researchers will test a cell-specific editing strategy that targets a metabolic pathway shared by many RP-causing genes, with safety steps for potential future human use. The work builds on strong early data and seeks to move toward a therapy that preserves cone function even as rods are lost. If successful, this could slow vision loss and improve quality of life for RP patients.
Why it matters
Potential benefit: If successful, the therapy could help preserve cone function and vision for longer in people with RP.
How similar studies have gone: Previous studies show that altering retinal metabolism can influence cone survival, but this exact cell-specific CRISPR approach in RP is investigational.
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.
-
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.
-
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.
-
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.
-
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.
-
Understanding and Treating Inherited Retinal Degeneration to Preserve Vision
A clinician-scientist at Stanford is pursuing gene-agnostic therapies to slow or prevent vision loss from inherited retinal diseases like 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.