How the FUS protein clumps in ALS and related dementia
Residue-by-residue details of FUS protein phase separation and aggregation
['FUNDING_R01'] · BROWN UNIVERSITY · NIH-11376301
This project looks at how tiny parts of the FUS protein make it stick together and form harmful clumps that can contribute to ALS and frontotemporal dementia.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | BROWN UNIVERSITY (nih funded) |
| Locations | 1 site (PROVIDENCE, UNITED STATES) |
| Trial ID | NIH-11376301 on ClinicalTrials.gov |
What this research studies
Researchers will map the individual building blocks (amino acid residues) across the FUS protein to find which pieces drive liquid-like clustering versus irreversible aggregation. They will use biochemical and biophysical tests plus cell-based experiments to watch how normal and mutated FUS behave under different conditions. By comparing dynamic, reversible assemblies to the solid inclusions linked to disease, the team aims to pinpoint molecular triggers of pathological clumping. That detailed molecular picture could guide later efforts to design drugs or interventions that prevent or reverse harmful aggregation.
Who could benefit from this research
Good fit: People with ALS or frontotemporal dementia caused by FUS mutations, or those willing to provide patient samples for related research, would be the most directly relevant candidates for follow-up studies.
Not a fit: Patients whose disease is unrelated to FUS biology or those seeking immediate treatment are unlikely to receive direct benefit from this laboratory-focused project.
Why it matters
Potential benefit: If successful, the work could identify specific molecular targets for therapies to stop or reduce harmful FUS aggregation in people with ALS or FTD.
How similar studies have performed: Laboratory research has previously shown that FUS can phase-separate and form aggregates, but residue-level mechanisms and the precise triggers of pathological aggregation remain largely novel and are the focus here.
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.
Conditions: Amyotrophic Lateral Sclerosis Motor Neuron Disease