Investigating how a specific immune pathway in neurons affects ALS and frontotemporal dementia.
Official title: Resolving the Role of Neuronal STING in Amyotrophic Lateral Sclerosis and Frontotemporal Dementia
Funded at Massachusetts General Hospital · NIH-10606865 · Data updated 2026-11-12
This study is looking at how a specific pathway in brain cells might be linked to diseases like ALS and frontotemporal dementia, focusing on how damage to certain genes can trigger this pathway and possibly lead to nerve cell problems, with the hope of finding new ways to help people with these conditions.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Massachusetts General Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | January 5, 2023 |
| Project end | December 31, 2025 |
| Funding year | 2023 |
| Award (this year) | $2,085,362 |
| NIH project number | 1RF1NS127407-01A1 |
| Project ID | NIH-10606865 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals diagnosed with ALS or frontotemporal dementia, particularly those with genetic links to the C9orf72 gene.
✕Less likely to help
Patients with neurodegenerative diseases not related to ALS or frontotemporal dementia may not benefit from this research.
What this research studies
This research explores the role of the STING pathway in neurons and its impact on neurodegenerative diseases like amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The study focuses on how DNA damage, particularly from the C9orf72 gene and TDP-43 protein, activates STING in neurons, potentially leading to neurodegeneration. By examining human brain samples and using advanced models, the researchers aim to understand the relationship between STING activation and disease progression. This could provide insights into new therapeutic targets for these conditions.
Why it matters
Potential benefit: If successful, this research could lead to new treatments that slow down or prevent the progression of ALS and frontotemporal dementia.
How similar studies have gone: While the role of STING in myeloid cells has been studied, this investigation into neuronal STING is relatively novel and untested.
Where this research is happening
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Massachusetts General Hospital
Boston, United States Active
Researchers
- Principal investigator: Wainger, Brian Jason — Massachusetts General Hospital
- Study coordinator: Wainger, Brian Jason
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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C9orf72 ALS/FTD Prevent: prepping for future treatment
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.
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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.
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.