Understanding cell-cleanup problems in C9ORF72-related ALS/FTD
Official title: Unraveling Mechanisms Governing Cell Clearance Deficits in C9ORF72-ALS/FTD
Funded at University of Missouri-Columbia · NIH-11606636 · Data updated 2026-11-12
Researchers are studying why the body's cell-cleanup systems fail in a genetic form of ALS/FTD to pave the way for better treatments.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Missouri-Columbia NIH-funded |
| Lab location | 1 site (Columbia, United States) |
| Project start | September 24, 2026 |
| Project end | August 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $539,427 |
| NIH project number | 1R56NS140507-01A1 |
| Project ID | NIH-11606636 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with C9ORF72-related ALS/FTD or their cells used for research, particularly those contributing to iMNs (induced motor neurons) models.
✕Less likely to help
Individuals without C9ORF72-related disease likely may not benefit from this specific mechanism-focused research.
What this research studies
This project uses patient-derived nerve cells to look at how autophagy and related protein disposal pathways break down in C9ORF72-ALS/FTD. By focusing on TBK1 and other players in the autophagosome formation process, the study aims to identify where the cleanup process stalls. The results could point to new targets to restore proper protein clearance and slow disease progression. The work combines human-derived cells with molecular analyses to translate findings into potential therapies for patients.
Why it matters
Potential benefit: If successful, this work could reveal targets to improve cell-cleanup and slow ALS/FTD progression, informing future therapies for patients.
How similar studies have gone: Previous work shows that protein clearance pathways are altered in ALS/FTD and that targeting these pathways can modify disease-relevant processes in cell models.
Where this research is happening
-
University of Missouri-Columbia
Columbia, United States Active
Researchers
- Principal investigator: Saxena, Smita — University of Missouri-Columbia
- Study coordinator: Saxena, Smita
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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.