Shared disease pathways in nerve cells from people with frontotemporal dementia
Official title: Investigating Common Molecular Pathogenic Pathways in Frontotemporal Dementia Patients iPSC-Derived Neurons
Funded at Univ of Massachusetts Med Sch Worcester · NIH-11296109 · Data updated 2026-11-12
This project uses nerve cells made from people with frontotemporal dementia to find common ways the cells break down that might point to new treatments.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Univ of Massachusetts Med Sch Worcester NIH-funded |
| Lab location | 1 site (Worcester, United States) |
| Project start | December 18, 2007 |
| Project end | January 31, 2031 |
| Funding year | 2026 |
| Award (this year) | $637,775 |
| NIH project number | 2R01NS057553-19 |
| Project ID | NIH-11296109 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with frontotemporal dementia or ALS, especially those with known C9ORF72 or TARDBP mutations or diagnosed sporadic FTD/ALS who can provide samples, would be the most relevant candidates to contribute to this work.
✕Less likely to help
People without FTD or ALS, or patients seeking an immediate clinical treatment benefit, are unlikely to benefit directly from participation in this basic/translational research.
What this research studies
Researchers grow cortical neurons from patients' cells that have been reprogrammed into iPSCs and compare cells from people with known genetic mutations (like C9ORF72 and TARDBP) and from sporadic FTD/ALS cases. They use gene-editing (CRISPR), gene-expression (transcriptomic) analyses, and complementary fly models to look for shared problems such as DNA damage and pathways that change disease severity. The goal is to identify molecular targets that could be tested for therapies in the future. The work combines patient-derived human cells with animal experiments to strengthen findings across systems.
Why it matters
Potential benefit: If successful, this work could reveal shared molecular targets across forms of FTD and ALS that guide development of new therapies.
How similar studies have gone: Patient-derived iPSC neuron models and Drosophila studies have previously revealed disease mechanisms in neurodegeneration, but translating those findings into effective treatments has been limited so far.
Where this research is happening
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Univ of Massachusetts Med Sch Worcester
Worcester, United States Active
Researchers
- Principal investigator: Gao, Fen-Biao — Univ of Massachusetts Med Sch Worcester
- Study coordinator: Gao, Fen-Biao
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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.