Repairing harmful genetic mutations in mitochondrial RNA
Official title: Engineering of PPR base editors to repair pathogenic SNPs at the level of RNA
Funded at California State University Los Angeles · NIH-10359636 · Data updated 2026-11-12
This study is exploring a new way to fix genetic mistakes in mitochondrial RNA that cause diseases like Leigh syndrome, using specially designed plant proteins to help repair these issues, which could lead to better treatments for people with these genetic disorders.
Quick facts
| Grant type | R15 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | California State University Los Angeles NIH-funded |
| Lab location | 1 site (Los Angeles, United States) |
| Project start | September 20, 2021 |
| Project end | August 31, 2025 |
| Funding year | 2021 |
| Award (this year) | $438,000 |
| NIH project number | 1R15GM144905-01 |
| Project ID | NIH-10359636 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research would be individuals diagnosed with Leigh syndrome or other mitochondrial disorders caused by specific T-to-C SNPs.
✕Less likely to help
Patients with non-mitochondrial genetic disorders or those without specific pathogenic SNPs may not benefit from this research.
What this research studies
This research focuses on developing a new method to correct specific genetic mutations in mitochondrial RNA that are linked to diseases like Leigh syndrome. The approach involves engineering proteins from plants that can recognize and edit these mutations at the RNA level, which is particularly challenging due to the unique structure of mitochondrial genomes. By modifying certain amino acids in these proteins, the researchers aim to enhance their ability to target and repair pathogenic SNPs effectively. This innovative technique could lead to significant advancements in treating genetic disorders caused by mitochondrial mutations.
Why it matters
Potential benefit: If successful, this research could provide a novel therapeutic approach for patients with mitochondrial diseases, potentially improving their health outcomes.
How similar studies have gone: While the approach of using engineered proteins for RNA editing is innovative, similar techniques have shown promise in other contexts, suggesting potential for success in this novel application.
Where this research is happening
-
California State University Los Angeles
Los Angeles, United States Active
Researchers
- Principal investigator: Hayes, Michael Lloyd — California State University Los Angeles
- Study coordinator: Hayes, Michael Lloyd
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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.