Understanding FUS-related ALS/FTD and how to fix it
Official title: Multimodal analysis and therapeutic targeting of aberrant nucleocytoplasmic compartmentalization in FUS-related neurodegenerative disease
Funded at Univ of Massachusetts Med Sch Worcester · NIH-11452976 · Data updated 2026-11-12
This NIH grant explores how problems with a key RNA-binding protein called FUS contribute to ALS and related diseases, using human cells and mouse models to uncover potential treatments.
Quick facts
| Grant type | R01 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 | May 15, 2019 |
| Project end | June 30, 2031 |
| Funding year | 2026 |
| Award (this year) | $1,123,020 |
| NIH project number | 2R01NS108769-06A1 |
| Project ID | NIH-11452976 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with ALS or related neurodegenerative conditions involving FUS mutations or dysfunction who might later participate in targeted therapies or biomarker studies.
✕Less likely to help
People without ALS/FTD or without FUS-related pathology may not directly benefit from this research.
What this research studies
Researchers will study how FUS mutations disrupt cellular transport and RNA processing using advanced imaging, biochemistry, structure, and genomics in human stem cell–derived neurons and mice. They will compare the effects of FUS mutation versus depletion, and test interventions like antisense oligonucleotides and CRISPR interference to see if correcting FUS problems can restore normal cell function. The goal is to map the exact defects caused by FUS dysfunction and identify points where therapies could intervene. This project emphasizes models that use human cells derived from patients and validated mouse systems to translate findings toward patient care.
Why it matters
Potential benefit: If successful, this work could point to new therapeutic strategies to slow or halt ALS/FTD in people with FUS-related disease.
How similar studies have gone: Prior studies in FUS biology and nucleocytoplasmic transport have shown promising mechanistic insights, supporting the potential for therapeutic targets in this area.
Where this research is happening
-
Univ of Massachusetts Med Sch Worcester
Worcester, United States Active
Researchers
- Principal investigator: Bosco, Daryl Angela — Univ of Massachusetts Med Sch Worcester
- Study coordinator: Bosco, Daryl Angela
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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.