New DNA-binding proteins to stop repeat instability in Huntington’s and related diseases
Official title: Targeting repeat expansion instability with de novo DNA slip-out binding proteins
Funded at Rice University · NIH-11353177 · Data updated 2026-11-12
Aiming to slow or stop the progression of diseases caused by DNA repeat expansions by creating new protein tools that stabilize the genome in patients.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Rice University NIH-funded |
| Lab location | 1 site (Houston, United States) |
| Project start | August 22, 2026 |
| Project end | July 31, 2029 |
| Funding year | 2026 |
| Award (this year) | $246,208 |
| NIH project number | 1R21EB039470-01 |
| Project ID | NIH-11353177 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with repeat expansion disorders such as Huntington’s disease, myotonic dystrophy, or spinocerebral ataxias.
✕Less likely to help
Individuals without REDs or without access to relevant delivery methods may not benefit.
What this research studies
This project plans to develop gene therapy tools that bind to DNA structures formed by repeating sequences, with the goal of preventing somatic instability that drives disease progression. Researchers will design and test de novo DNA-binding proteins that recognize nonstandard DNA shapes, potentially stopping harmful repeat expansion in cells. The work will start with models related to Huntington’s disease and extend to other repeat expansion diseases, using advanced delivery methods to target affected tissues. If successful, these tools could complement or reduce disease advancement by keeping repeat lengths stable over time.
Why it matters
Potential benefit: If successful, patients could experience slower disease progression or delayed onset due to stabilized genetic repeats.
How similar studies have gone: Preliminary work in repeat instability suggests that targeting DNA structure is a promising but still early approach.
Where this research is happening
-
Rice University
Houston, United States Active
Researchers
- Principal investigator: Glasscock, Cameron — Rice University
- Study coordinator: Glasscock, Cameron
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.