T cell roles in a rare juvenile ALS form (SETX-related)
Official title: T cell Immunity in a Rare Juvenile Form of Motor Neuron Disease
Funded at University of California-Irvine · NIH-11395430 · Data updated 2026-11-12
A study to understand how immune cells, especially CD8 T cells, influence a rare juvenile form of ALS caused by SETX mutations.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California-Irvine NIH-funded |
| Lab location | 1 site (Irvine, United States) |
| Project start | September 1, 2023 |
| Project end | August 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $637,482 |
| NIH project number | 5R01NS123287-04 |
| Project ID | NIH-11395430 on NIH RePORTER |
Who could benefit from this research
✓Could help
Individuals with ALS4 or SETX-related juvenile ALS, and possibly other forms of ALS, who may contribute samples or participate in future trials.
✕Less likely to help
Patients with non-SETX ALS or unrelated neurodegenerative conditions may not directly benefit from this line of investigation.
What this research studies
This project looks at how the immune system, including CD8 T cells in the brain and blood, may contribute to disease onset and progression in ALS4. Using a mouse model and patient samples, researchers will test what happens when CD8 T cells are transferred or removed and examine immune and brain tissue to map interactions. They will analyze gene expression networks to identify pathways linking immune activity to neurodegeneration and validate findings in both mice and people with ALS. The goal is to uncover immune-related mechanisms that could be targeted for future therapies in juvenile ALS patients.
Why it matters
Potential benefit: Understanding immune involvement could lead to new treatment strategies that slow or modify disease in young people with ALS4.
How similar studies have gone: Previous work shows immune components can affect neurodegeneration, and this study extends that to a specific juvenile ALS form with potential translational relevance.
Where this research is happening
-
University of California-Irvine
Irvine, United States Active
Researchers
- Principal investigator: Marazzi, Ivan — University of California-Irvine
- Study coordinator: Marazzi, Ivan
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T cells in a rare juvenile form of ALS (ALS4)
This research looks at whether certain CD8 T immune cells are linked to ALS4, a rare juvenile form of ALS, by studying mouse models and samples from people with ALS.
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.