Exploring new ways to treat Huntington's disease by targeting genetic changes.
Official title: Modulation of Somatic Repeat Expansion as a Therapeutic Approach to Huntington's Disease
Funded at Univ of Massachusetts Med Sch Worcester · NIH-11056772 · Data updated 2026-11-12
This study is looking at how certain genetic changes in Huntington's disease affect its progression, and it hopes to find new ways to help patients by exploring treatments that could lessen the impact of these changes.
Quick facts
| Grant type | Fellowship grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Univ of Massachusetts Med Sch Worcester NIH-funded |
| Lab location | 1 site (Worcester, United States) |
| Project start | April 1, 2023 |
| Project end | July 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $33,758 |
| NIH project number | 5F31NS132424-03 |
| Project ID | NIH-11056772 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Huntington's disease, particularly those experiencing early symptoms.
✕Less likely to help
Patients with other neurodegenerative disorders unrelated to Huntington's disease may not receive any benefit from this research.
What this research studies
This research focuses on Huntington's disease, which is linked to specific genetic changes in the huntingtin gene. It aims to understand how these genetic changes, particularly the expansion of CAG repeats, contribute to the disease's progression. By investigating the role of mismatch repair proteins in this process, the study seeks to develop new therapeutic strategies using small interfering RNA and antisense oligonucleotides to potentially reduce the harmful effects of these genetic expansions. Patients may benefit from insights that could lead to more effective treatments for Huntington's disease.
Why it matters
Potential benefit: If successful, this research could lead to innovative therapies that slow down or alter the progression of Huntington's disease.
How similar studies have gone: While the approach of targeting genetic mechanisms in Huntington's disease is being explored, this specific methodology involving mismatch repair proteins is relatively novel and has not been extensively tested.
Where this research is happening
-
Univ of Massachusetts Med Sch Worcester
Worcester, United States Active
Researchers
- Principal investigator: Belgrad, Jillian — Univ of Massachusetts Med Sch Worcester
- Study coordinator: Belgrad, Jillian
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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.