Understanding how TBK1 affects lysosomes in neurodegenerative diseases
Lysosomal Regulation and Functions for TBK1 in Neurodegenerative Diseases
This study is looking at how a protein called TBK1, which is important for brain health, helps keep our cells clean and how changes in this protein and another one can lead to conditions like ALS and frontotemporal dementia, with the hope of finding new ways to treat these diseases.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Yale University NIH-funded |
| Lab location | 1 site (New Haven, United States) |
| Project ID | NIH-11165602 on NIH RePORTER |
What this research studies
This research investigates the role of TBK1, a protein linked to neurodegenerative diseases, in regulating lysosomes, which are essential for clearing harmful materials from cells. The study focuses on how mutations in TBK1 and another gene, C9orf72, contribute to conditions like amyotrophic lateral sclerosis and frontotemporal dementia. By exploring the molecular mechanisms that activate TBK1 at lysosomes, the research aims to uncover new therapeutic targets for these diseases. Patients may benefit from insights that could lead to innovative treatments for neurodegenerative disorders.
Who could benefit from this research
Good fit: Ideal candidates for this research include individuals diagnosed with amyotrophic lateral sclerosis, frontotemporal dementia, or related neurodegenerative conditions.
Not a fit: Patients with neurodegenerative diseases not linked to TBK1 or C9orf72 mutations may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new therapies that improve outcomes for patients with neurodegenerative diseases.
How similar studies have performed: Previous research has shown promising results in targeting lysosomal functions in neurodegenerative diseases, indicating potential for success in this area.
Where this research is happening
New Haven, United States
- Yale University — New Haven, United States (Active)
Researchers
- Principal investigator: Ferguson, Shawn — Yale University
- Study coordinator: Ferguson, Shawn
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.