Targeting MSUT2 with small molecules to slow tau-related dementia
Official title: Targeting MSUT2 with small molecules to ameliorate pathological tau
Funded at Seattle Inst for Biomedical/clinical Res · NIH-11463459 · Data updated 2026-11-12
Aims to test whether small molecules that block MSUT2’s interaction with RNA can slow or reverse tau-related brain damage in people affected by Alzheimer’s disease and related dementias.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Seattle Inst for Biomedical/clinical Res NIH-funded |
| Lab location | 1 site (Seattle, United States) |
| Project start | September 1, 2023 |
| Project end | August 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $688,382 |
| NIH project number | 4R01AG078335-02 |
| Project ID | NIH-11463459 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults diagnosed with Alzheimer’s disease dementia or related tauopathies who may participate in future biomarker or early-phase therapeutic studies.
✕Less likely to help
Individuals without tau pathology or those who do not respond to MSUT2-targeted interventions may not experience benefit.
What this research studies
This project focuses on tau pathology, a key driver of Alzheimer’s disease and related dementias. It builds on prior work showing MSUT2’s role in tau toxicity and identifies drug-like compounds that disrupt MSUT2’s binding to poly(A) RNA. Through high-throughput screening and preclinical studies, researchers will pursue small molecules that may modify disease progression by targeting tau pathology. If successful, these compounds could become starting points for therapies aiming to slow neurodegeneration in patients with AD and ADRD.
Why it matters
Potential benefit: If successful, the approach could slow or reverse tau-driven neurodegeneration in patients with Alzheimer’s disease and related dementias.
How similar studies have gone: Prior tau-targeted approaches have shown mixed results, but identifying MSUT2 inhibitors is a novel strategy with encouraging preclinical signals.
Where this research is happening
-
Seattle Inst for Biomedical/clinical Res
Seattle, United States Active
Researchers
- Principal investigator: Kraemer, Brian C. — Seattle Inst for Biomedical/clinical Res
- Study coordinator: Kraemer, Brian C.
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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.