Improving cone cell survival in retinitis pigmentosa by altering metabolism
Official title: Reprogramming the metabolome to overcome the genetic heterogeneity in retinitis pigmentosa
Funded at Columbia University Health Sciences · NIH-10900808 · Data updated 2026-11-12
This study is looking at ways to help protect the vision of people with retinitis pigmentosa by changing how certain cells in the eye get their energy, using special techniques in mice to find new treatments that could prevent blindness.
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 | August 1, 2020 |
| Project end | July 31, 2025 |
| Funding year | 2024 |
| Award (this year) | $405,000 |
| NIH project number | 5R01EY031354-05 |
| Project ID | NIH-10900808 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with retinitis pigmentosa, particularly those experiencing early to mid-stage vision loss.
✕Less likely to help
Patients with advanced retinitis pigmentosa who have already lost significant vision may not benefit from this research.
What this research studies
This research focuses on retinitis pigmentosa (RP), a genetic condition that leads to blindness due to the death of photoreceptor cells in the retina. The project aims to understand how cone cells, which are crucial for vision, can be protected from dying by reprogramming their metabolism. Researchers will use advanced techniques, including CRISPR, to manipulate the metabolic processes in both cone cells and retinal pigment epithelium (RPE) cells in mouse models of RP. By enhancing or suppressing specific metabolic pathways, the study seeks to find new therapies that could prevent vision loss in patients with RP.
Why it matters
Potential benefit: If successful, this research could lead to new treatments that preserve vision in patients with retinitis pigmentosa.
How similar studies have gone: While the approach of reprogramming metabolism in retinal cells is innovative, similar strategies in other conditions have shown promise, suggesting potential for success.
Where this research is happening
-
Columbia University Health Sciences
New York, United States Active
Researchers
- Principal investigator: Wang, Nan-Kai — Columbia University Health Sciences
- Study coordinator: Wang, Nan-Kai
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.
-
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.