Investigating how small molecules can help treat Huntington's disease
Official title: Understanding small molecule modulation of splicing for Huntington's disease therapy
Funded at University of Colorado Denver · NIH-11127253 · Data updated 2026-11-12
This study is looking at a new way to help people with Huntington's disease by using special molecules that can help reduce the harmful huntingtin protein in their bodies, which could lead to better treatment options for managing the condition.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Colorado Denver NIH-funded |
| Lab location | 1 site (Aurora, UNITED STATES) |
| Project start | April 1, 2025 |
| Project end | March 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $195,000 |
| NIH project number | 1R21NS142950-01 |
| Project ID | NIH-11127253 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Huntington's disease who are seeking new treatment options.
✕Less likely to help
Patients with other neurological disorders unrelated to Huntington's disease may not benefit from this research.
What this research studies
This research focuses on using small molecule splicing modulators to reduce levels of the harmful huntingtin protein in patients with Huntington's disease. By inducing the inclusion of a specific 'poison' exon in the huntingtin mRNA, the study aims to promote its degradation and decrease the protein's harmful effects. The research will explore the mechanisms of these modulators to optimize their effectiveness and minimize side effects, particularly in the central nervous system. Patients may benefit from a new therapeutic approach that could lead to improved treatment options for Huntington's disease.
Why it matters
Potential benefit: If successful, this research could lead to a novel therapy that significantly reduces the harmful effects of Huntington's disease.
How similar studies have gone: Previous research has shown promise with similar approaches in treating other central nervous system diseases, indicating potential for success in this area as well.
Where this research is happening
-
University of Colorado Denver
Aurora, United States Active
Researchers
- Principal investigator: Zhao, Rui — University of Colorado Denver
- Study coordinator: Zhao, Rui
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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.