Subcellular clues to why some brain neurons get hit 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-11463408 · Data updated 2026-11-12
This research looks at tiny cell parts in brain neurons to understand why certain neurons are more damaged in ALS and FTD, using mouse models and human-like brain cell systems.
Quick facts
| Grant type | R01 grant |
|---|---|
| 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, 2028 |
| Funding year | 2026 |
| Award (this year) | $665,314 |
| NIH project number | 4R01AG083085-02 |
| Project ID | NIH-11463408 on NIH RePORTER |
Who could benefit from this research
✓Could help
Individuals with ALS or FTD or those at risk may benefit from future therapies informed by these mechanisms.
✕Less likely to help
People not affected by ALS or FTD or those not involved in related future trials may not directly benefit.
What this research studies
Researchers will study neuron subtypes in ALS and FTD to identify specific RNA, protein, and translation patterns that make some neurons vulnerable. They will use new subcellular methods in mouse models and human cortico-spinal assembloids to compare affected neurons with related but more resistant ones. The goal is to uncover precise molecular differences that could point to new therapeutic targets. The project focuses on corticospinal neurons and related neuron types involved in movement and cognition, seeking mechanisms that explain selective degeneration.
Why it matters
Potential benefit: Insights from this work could guide the development of therapies that protect vulnerable neurons in ALS and FTD.
How similar studies have gone: Previous studies have started to reveal subtype-specific vulnerabilities, but this project aims for deeper subcellular insights that are still being explored.
Where this research is happening
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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.