Lowering mutant huntingtin in aging brains affected by Huntington's disease
Understanding the mechanisms that modulate the effects of mutant Huntingtin lowering in aging Huntington's disease model mice
This work looks at how reducing the disease-causing huntingtin protein changes brain health in models of Huntington's disease, focusing on effects that appear with aging.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Virginia NIH-funded |
| Lab location | 1 site (Charlottesville, United States) |
| Project ID | NIH-11264914 on NIH RePORTER |
What this research studies
From my perspective as someone affected by Huntington's disease, the team uses genetically controlled mice that let them reduce either the mutant or total huntingtin protein at different ages and measure behavior and brain changes. They compare early-life (~50% before symptoms) versus later-life lowering to see which timing works best and whether lowering total huntingtin causes harm. The researchers study inflammation, astrocyte responses, and other brain mechanisms to explain differences and link their findings to antisense oligonucleotide and related therapies being tested in people.
Who could benefit from this research
Good fit: The findings would most directly inform future trials for adults with early-stage Huntington's disease or pre-symptomatic carriers of the HTT mutation.
Not a fit: People with very advanced, late-stage Huntington's disease may not benefit from timing insights derived from these aged mouse models.
Why it matters
Potential benefit: If successful, this work could help doctors pick safer timing and targets for huntingtin-lowering treatments for people with Huntington's disease.
How similar studies have performed: Earlier animal experiments and ongoing antisense oligonucleotide trials have shown promise for lowering huntingtin, but long-term effects in aged models are less well tested.
Where this research is happening
Charlottesville, United States
- University of Virginia — Charlottesville, United States (Active)
Researchers
- Principal investigator: Zeitlin, Scott — University of Virginia
- Study coordinator: Zeitlin, Scott
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 below.