How parts of the FUS protein clump in ALS and related dementia
Residue-by-residue details of FUS protein phase separation and aggregation
Researchers are mapping which bits of the FUS protein stick together and form clumps that may damage nerves in people with ALS or frontotemporal dementia.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Brown University NIH-funded |
| Lab location | 1 site (Providence, United States) |
| Project ID | NIH-11176250 on NIH RePORTER |
What this research studies
If you have ALS or frontotemporal dementia, this work looks at how individual building blocks of the FUS protein stick together and form harmful clumps. Lab teams will change single amino acids and use purified proteins, cell models, and high-resolution imaging and biochemical tests to map which residues drive reversible liquid-like assemblies versus irreversible aggregates. They will also examine FUS versions linked to familial ALS/FTD to see how disease mutations alter behavior. The goal is to pinpoint exact molecular interactions that could be blocked by future drugs or therapies.
Who could benefit from this research
Good fit: Ideal participants would include people with ALS or frontotemporal dementia—especially those with known FUS mutations—who can provide samples or participate in related studies.
Not a fit: People with other forms of ALS without FUS involvement, or those needing immediate clinical therapy, are unlikely to gain direct short-term benefit from this basic lab work.
Why it matters
Potential benefit: This could point to precise molecular targets to stop or reverse toxic FUS clumps in ALS and related dementias.
How similar studies have performed: Previous lab work has shown RNA-binding proteins like FUS can form liquid droplets and pathological aggregates, but translating these findings into effective treatments is still largely unproven.
Where this research is happening
Providence, United States
- Brown University — Providence, United States (Active)
Researchers
- Principal investigator: Fawzi, Nicolas Lux — Brown University
- Study coordinator: Fawzi, Nicolas Lux
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.