Mapping harmful protein tagging in C9orf72-linked ALS and FTD
Multiplexed Quantification of the Protein Poly-ADP-Ribosylation Landscape in C9ORF72-ALS/FTD
This project will map a protein modification called PARylation in C9orf72-related ALS and frontotemporal dementia to learn how it harms nerve cells.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Columbia University Health Sciences NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project ID | NIH-11370239 on NIH RePORTER |
What this research studies
If you take part, researchers will use a new multiplexed measurement method to find which proteins become PARylated in C9orf72-linked ALS and FTD using cells, animal models, and patient-derived samples. They will apply sensitive lab tools like mass spectrometry and molecular assays to pinpoint where PARylation occurs and how it may trigger neuron damage. The team will test whether lowering PARylation or blocking the PARP1 enzyme protects neurons in experimental models. Results will guide which molecules or pathways could be targeted in future treatments or clinical trials.
Who could benefit from this research
Good fit: Ideal candidates would be people with ALS or FTD who have a confirmed C9orf72 repeat expansion or who are willing to provide blood, tissue, or clinical information for research.
Not a fit: People without ALS or FTD, or those with unrelated conditions, are unlikely to gain direct benefit from this project.
Why it matters
Potential benefit: If successful, this work could reveal new drug targets to slow or prevent nerve-cell loss in C9orf72-associated ALS/FTD.
How similar studies have performed: Prior cell and animal studies show that PARP1 inhibition can protect neurons in models of ALS, FTD, and other dementias, but a comprehensive PARylation map in C9orf72 cases is novel.
Where this research is happening
New York, United States
- Columbia University Health Sciences — New York, United States (Active)
Researchers
- Principal investigator: Yu, Yonghao — Columbia University Health Sciences
- Study coordinator: Yu, Yonghao
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 below.