Using RNA helicases to address issues caused by repeat expansion disorders
Official title: RNA helicases to combat RNA phase transitions in repeat expansion disorders
Funded at University of Pennsylvania · NIH-11012250 · Data updated 2026-11-12
This study is looking at how certain proteins called RNA helicases can help reduce the harmful effects of rare genetic disorders that affect the brain, by figuring out how they can stop toxic RNA from forming in cells.
Quick facts
| Grant type | Fellowship grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Pennsylvania NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | December 1, 2023 |
| Project end | June 30, 2026 |
| Funding year | 2025 |
| Award (this year) | $69,080 |
| NIH project number | 5F32NS131218-02 |
| Project ID | NIH-11012250 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with repeat expansion disorders, particularly those with severe symptoms or early onset.
✕Less likely to help
Patients with non-repeat expansion disorders or those who do not exhibit significant neurological symptoms may not benefit from this research.
What this research studies
This research investigates the role of RNA helicases in managing the harmful effects of repeat expansion disorders, which are rare diseases affecting the central nervous system. These disorders are caused by genetic repeat expansions that lead to toxic RNA and protein products. The study aims to understand how RNA helicases can influence the formation of toxic RNA foci in cells, potentially offering a new therapeutic approach. By exploring the mechanisms of RNA phase transitions, the research seeks to identify ways to mitigate the associated cellular toxicity.
Why it matters
Potential benefit: If successful, this research could lead to new treatments for patients suffering from repeat expansion disorders, improving their quality of life.
How similar studies have gone: While the specific approach of targeting RNA helicases in repeat expansion disorders is novel, similar strategies have shown promise in other neurological conditions.
Where this research is happening
-
University of Pennsylvania
Philadelphia, United States Active
Researchers
- Principal investigator: Miller, Linamarie — University of Pennsylvania
- Study coordinator: Miller, Linamarie
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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.