Editing RNA to correct genetic mutations
Official title: High-throughput screening and structure-guided optimization of oligonucleotides for site-directed RNA editing by ADARs.
Funded at University of California at Davis · NIH-11051210 · Data updated 2026-11-12
This study is exploring a new way to fix genetic mutations that cause diseases by using a special enzyme to change certain parts of RNA, which could help your body make the right proteins it needs to stay healthy.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California at Davis NIH-funded |
| Lab location | 1 site (Davis, United States) |
| Project start | April 1, 2023 |
| Project end | March 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $309,993 |
| NIH project number | 5R01GM149799-03 |
| Project ID | NIH-11051210 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals with genetic disorders resulting from specific adenosine-to-guanosine mutations in their RNA.
✕Less likely to help
Patients with genetic disorders not caused by point mutations or those whose mutations do not involve adenosine bases may not benefit from this research.
What this research studies
This research focuses on using a specialized enzyme called ADAR to edit RNA sequences in order to correct specific genetic mutations that cause diseases. By targeting adenosine bases in RNA transcripts, the goal is to convert them into guanosine, which can help produce functional proteins instead of truncated ones. The approach involves designing guide oligonucleotides that can direct the editing process to specific locations in the RNA, particularly where harmful mutations occur. A high-throughput screening method will be employed to identify the most effective oligonucleotides for this purpose.
Why it matters
Potential benefit: If successful, this research could lead to new treatments for genetic disorders caused by point mutations.
How similar studies have gone: Previous research has shown promise in using RNA editing technologies for genetic corrections, indicating potential success for this approach.
Where this research is happening
-
University of California at Davis
Davis, United States Active
Researchers
- Principal investigator: Fisher, Andrew J — University of California at Davis
- Study coordinator: Fisher, Andrew J
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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.