Axon-guidance genes and ALS/FTD: how neuron paths go wrong
Official title: Axon guidance deregulation in ALS and frontotemporal dementia
Funded at University of Wisconsin-Madison · NIH-11458497 · Data updated 2026-11-12
A short-term NIH grant will explore how axon guidance genes contribute to ALS and frontotemporal dementia in people and neuronal cells.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Wisconsin-Madison NIH-funded |
| Lab location | 1 site (Madison, United States) |
| Project start | August 1, 2026 |
| Project end | July 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $427,625 |
| NIH project number | 1R21NS147268-01A1 |
| Project ID | NIH-11458497 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with ALS or FTD or those at risk may eventually benefit from therapies targeting UBQLN2-related pathways.
✕Less likely to help
This specific basic science study does not directly enroll patients or provide immediate treatment.
What this research studies
This project uses a “fly to human” approach to find how UBQLN2 mutations drive neurodegeneration in ALS and FTD, starting with fruit fly models and then testing findings in patient-derived motor neurons. The researchers will perform genetic screens to identify axon guidance pathways that modify UBQLN2 toxicity, aiming to uncover mechanisms that lead to neuron damage. By linking basic gene pathways to human neuron behavior, the study could point to new targets for therapies in ALS/FTD.
Why it matters
Potential benefit: If successful, the work could reveal new targets to slow or prevent neuron damage in ALS and FTD.
How similar studies have gone: Previous work shows UBQLN2 mutations promote protein aggregation and neurodegeneration in model systems, with multiple pathways still being explored.
Where this research is happening
-
University of Wisconsin-Madison
Madison, United States Active
Researchers
- Principal investigator: Tibbetts, Randal Scot — University of Wisconsin-Madison
- Study coordinator: Tibbetts, Randal Scot
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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.