Using self-amplifying RNAs to improve gene therapy for eye diseases
Official title: Utilizing circular, self-amplifying RNAs for prolonged expression
Funded at State University of New York at Buffalo · NIH-10975447 · Data updated 2026-11-12
This study is looking at a new way to use gene therapy to help people with inherited eye conditions by using special RNA techniques that might be safer and more effective than current methods, making it easier for patients to get the treatment they need.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | State University of New York at Buffalo NIH-funded |
| Lab location | 1 site (Amherst, United States) |
| Project start | September 30, 2024 |
| Project end | September 29, 2026 |
| Funding year | 2024 |
| Award (this year) | $346,500 |
| NIH project number | 1R21EY036603-01 |
| Project ID | NIH-10975447 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with inherited retinal dystrophies who are seeking innovative treatment options.
✕Less likely to help
Patients with retinal conditions that are not inherited or those who have already undergone irreversible damage to their retina may not benefit from this research.
What this research studies
This research investigates a new approach to gene therapy for inherited retinal dystrophies by utilizing self-amplifying and self-splicing RNA technologies. The goal is to develop a more effective and less invasive method for delivering corrective genes to retinal cells compared to traditional adeno-associated virus (AAV) methods. By exploring the longevity and potential side effects of these RNA technologies, the research aims to create a treatment that can provide longer-lasting effects without the need for destructive surgical procedures. This innovative approach could lead to more accessible and cost-effective therapies for patients with retinal diseases.
Why it matters
Potential benefit: If successful, this research could lead to safer and more effective gene therapies that improve vision for patients with inherited retinal dystrophies.
How similar studies have gone: Other research has shown promise in using self-amplifying RNA technologies for vaccine development, indicating potential for success in therapeutic applications as well.
Where this research is happening
-
State University of New York at Buffalo
Amherst, United States Active
Researchers
- Principal investigator: Farkas, Michael — State University of New York at Buffalo
- Study coordinator: Farkas, Michael
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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.