Investigating why certain neurons are more vulnerable in ALS and FTD
Official title: Subcellular mechanisms of subtype-specific neuron vulnerability in ALS and FTD: dysregulation of synapse-localized RNA, protein, and translation in mouse models and human cortico-spinal assembloids
Funded at Harvard University · NIH-10716562 · Data updated 2026-11-12
This study is looking at why some nerve cells are more likely to break down in conditions like ALS and frontotemporal dementia, using both mice and human brain models, to find clues that could help develop new treatments for these diseases.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Harvard University NIH-funded |
| Lab location | 1 site (Cambridge, United States) |
| Project start | September 1, 2023 |
| Project end | August 31, 2026 |
| Funding year | 2023 |
| Award (this year) | $2,001,864 |
| NIH project number | 1RF1AG083085-01 |
| Project ID | NIH-10716562 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with ALS or FTD, particularly those with familial forms of these diseases.
✕Less likely to help
Patients with other neurodegenerative diseases that do not involve the specific neuronal subtypes studied may not benefit from this research.
What this research studies
This research focuses on understanding the specific molecular mechanisms that make certain types of neurons particularly vulnerable to degeneration in amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). By examining both mouse models and human cortico-spinal assembloids, the study aims to identify differences in RNA, protein, and translation processes between affected and unaffected neurons. This could help uncover why only specific neuronal subtypes degenerate despite many neurons expressing similar genetic risk factors. The findings may lead to new insights into potential therapeutic targets for these conditions.
Why it matters
Potential benefit: If successful, this research could lead to targeted therapies that protect vulnerable neurons and improve outcomes for patients with ALS and FTD.
How similar studies have gone: Previous research has shown promise in understanding neurodegeneration through molecular mechanisms, but this specific approach is relatively novel.
Where this research is happening
-
Harvard University
Cambridge, United States Active
Researchers
- Principal investigator: Macklis, Jeffrey D — Harvard University
- Study coordinator: Macklis, Jeffrey D
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.