Lowering STAU1 to help ALS and related TDP-43 nerve diseases
Targeting STAU1 for TDP-43 proteinopathies
This project develops antisense drugs to reduce the STAU1 protein for people with ALS and other TDP-43 protein problems.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Utah State Higher Education System--University of Utah NIH-funded |
| Lab location | 1 site (Salt Lake City, United States) |
| Project ID | NIH-11146328 on NIH RePORTER |
What this research studies
Researchers are creating antisense oligonucleotides (ASOs) that lower a protein called STAU1, which is too abundant in many ALS and TDP-43 disease models. They have already found ASOs that cut STAU1 levels by 90-99% in patient-derived cells and can target mouse Stau1 for animal testing. The team will optimize ASO sequence and chemistry, test the best candidates in animal disease models, and collect data needed to advance toward human testing. The work builds on related ASO efforts (like ATXN2-targeting drugs) that have moved into early clinical trials.
Who could benefit from this research
Good fit: People with ALS or other disorders driven by TDP-43 proteinopathy, or patients willing to donate biological samples for ASO development, would be the most relevant candidates.
Not a fit: Patients whose disease is not related to TDP-43 proteinopathy, or those not eligible for future ASO trials, may not benefit from this work.
Why it matters
Potential benefit: If successful, this could yield a new antisense therapy that slows or improves symptoms in ALS and other TDP-43 proteinopathies.
How similar studies have performed: Antisense therapies targeting a related protein (ATXN2) have progressed to early human trials, while STAU1 targeting is a newer approach with promising preclinical results.
Where this research is happening
Salt Lake City, United States
- Utah State Higher Education System--University of Utah — Salt Lake City, United States (Active)
Researchers
- Principal investigator: Scoles, Daniel R — Utah State Higher Education System--University of Utah
- Study coordinator: Scoles, Daniel R
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.