Using advanced genome editing to treat inherited retinal diseases
Official title: Precision genome editing in vivo to treat retinal diseases
Funded at University of California-Irvine · NIH-11047716 · Data updated 2026-11-12
This study is exploring new ways to use advanced gene editing tools to help people with inherited eye disorders that can lead to blindness, aiming to safely fix the genetic issues causing their vision problems.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California-Irvine NIH-funded |
| Lab location | 1 site (Irvine, United States) |
| Project start | March 1, 2023 |
| Project end | February 28, 2026 |
| Funding year | 2025 |
| Award (this year) | $618,911 |
| NIH project number | 5R01EY034501-03 |
| Project ID | NIH-11047716 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with specific inherited retinal disorders caused by genetic mutations.
✕Less likely to help
Patients with retinal diseases not caused by genetic mutations or those with advanced stages of retinal degeneration may not benefit from this research.
What this research studies
This research focuses on developing innovative genome editing techniques to treat inherited retinal disorders, which are a major cause of blindness. The approach utilizes precision genome editing tools, such as adenine base editors and prime editing technology, to correct genetic mutations without causing significant damage to the DNA. By delivering these genome editors in a controlled manner, the research aims to improve the efficiency and safety of treatments for patients suffering from these conditions.
Why it matters
Potential benefit: If successful, this research could lead to effective treatments that restore vision for patients with inherited retinal diseases.
How similar studies have gone: Previous research has shown promising results using CRISPR technology for gene therapy, indicating potential success for this novel approach.
Where this research is happening
-
University of California-Irvine
Irvine, United States Active
Researchers
- Principal investigator: Palczewski, Krzysztof — University of California-Irvine
- Study coordinator: Palczewski, Krzysztof
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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.