Huntingtin's role in brain inflammation in Huntington's disease
Huntingtin links inflammasome pathway in Huntington's disease Pathogenesis
This project looks at whether the mutant huntingtin protein switches on the NLRP3 inflammasome in brain cells of people with Huntington's disease to find new treatment targets.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Johns Hopkins University NIH-funded |
| Lab location | 1 site (Baltimore, United States) |
| Project ID | NIH-11263669 on NIH RePORTER |
What this research studies
Researchers will examine brain tissue from people with Huntington's disease, lab-grown human striatal neurons, and mouse models to see if mutant huntingtin activates the NLRP3 inflammasome. They will study how this activation leads to neuron damage and inflammatory signaling. The team will use genetic knockdown of NLRP3 and other molecular tools in cells and animals to test whether blocking the inflammasome prevents degeneration. These lab-based findings aim to point toward therapies that could be tested in people.
Who could benefit from this research
Good fit: Ideal participants would be people with Huntington's disease or carriers of the HTT mutation who could donate tissue samples or be considered for future clinical trials based on these findings.
Not a fit: People without Huntington's disease or those seeking immediate symptom relief are unlikely to benefit directly from this laboratory-focused research.
Why it matters
Potential benefit: If successful, this work could identify new targets to reduce inflammation-driven neuron loss and lead to therapies that slow or stop Huntington's disease progression.
How similar studies have performed: Previous lab studies, including the team's pilot data, showed that mutant huntingtin can engage NLRP3 and that knocking down NLRP3 protected cultured neurons and improved outcomes in some models, but clinical benefits in people are unproven.
Where this research is happening
Baltimore, United States
- Johns Hopkins University — Baltimore, United States (Active)
Researchers
- Principal investigator: Jiang, Mali — Johns Hopkins University
- Study coordinator: Jiang, Mali
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.