C9orf72 ALS/FTD Prevent: prepping for future treatment
Official title: C9orf72 amyotrophic lateral sclerosis/frontotemporal dementia Prevent
Funded at University of Miami School of Medicine · NIH-11431628 · Data updated 2026-11-12
This grant aims to get ready for a preventive treatment for people who carry the C9orf72 genetic change that can lead to ALS or frontotemporal dementia.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Miami School of Medicine NIH-funded |
| Lab location | 1 site (Coral Gables, United States) |
| Project start | September 1, 2026 |
| Project end | August 31, 2031 |
| Funding year | 2026 |
| Award (this year) | $3,730,537 |
| NIH project number | 1R01AG103000-01 |
| Project ID | NIH-11431628 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals who carry the C9orf72 expansion and are pre-symptomatic for ALS/FTD.
✕Less likely to help
Individuals without the C9orf72 expansion or those already showing clinical ALS/FTD symptoms may not benefit.
What this research studies
The study focuses on people who carry the C9orf72 expansion but have not yet developed ALS or FTD. Researchers will examine clinical markers and biomarkers to predict when symptoms might begin and to guide early interventions. The project will evaluate factors such as thalamic volume, plasma neurofilament light, and personalized risk estimates to inform future prevention trials. It’s designed to identify the best ways to screen and monitor at‑risk individuals so that future therapies can be tested sooner.
Why it matters
Potential benefit: If successful, the work could speed the development of preventive therapies for people at genetic risk, potentially delaying or preventing disease onset.
How similar studies have gone: Previous work in other genetic neurodegenerative conditions has shown the value of biomarkers and early markers in informing prevention trials, but this specific prevention pathway is relatively novel.
Where this research is happening
-
University of Miami School of Medicine
Coral Gables, United States Active
Researchers
- Principal investigator: Benatar, Michael — University of Miami School of Medicine
- Study coordinator: Benatar, Michael
Related research
Other NIH-funded projects on the same topics.
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Investigating how DNA damage and repair mechanisms are altered in ALS and frontotemporal dementia.
This study is looking at how problems with DNA repair might contribute to conditions like ALS and frontotemporal dementia, and it’s testing ways to fix these issues in cells from patients to help develop new treatments.
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Understanding how a genetic repeat causes harmful protein production in ALS/FTD
Aimed at uncovering how a genetic repeat in the C9orf72 gene triggers abnormal protein production that damages nerve cells in ALS and FTD.
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How C9orf72 RNA repeats make toxic proteins in ALS and FTD
This research looks for how a repeated RNA sequence in the C9orf72 gene creates toxic proteins that can harm people with ALS or FTD.
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Cell and tissue changes that lead to sporadic ALS and frontotemporal dementia
This project looks at how physical changes inside cells and in their tissue can make TDP‑43 protein clump in people with sporadic ALS and frontotemporal dementia.
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Investigating new treatments for frontotemporal dementia and ALS using gene editing
This study is looking at ways to help people with frontotemporal dementia and ALS by using a special gene-editing tool to fix a harmful gene that causes these diseases.
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Understanding how certain genetic repeats cause neurodegenerative diseases
This study is looking into how a specific genetic change in the C9orf72 gene causes harmful proteins to form, which are linked to ALS and Frontotemporal Dementia, and it aims to understand how this happens in nerve cells to help find better ways to tackle these conditions.
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.