Investigating how TDP43 protein is broken down in ALS
TDP43 Degradation by the Lysosomal Proteases in Amyotrophic Lateral Sclerosis
This study is looking into how a protein called TDP43, which builds up in people with ALS, can be broken down by certain cell helpers, with the hope of finding new ways to protect motor neurons and improve treatments for ALS patients.
Quick facts
| Grant type | Career grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Francisco NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project ID | NIH-10843271 on NIH RePORTER |
What this research studies
This research focuses on understanding the mechanisms behind motor neuron loss in Amyotrophic Lateral Sclerosis (ALS), a severe neurodegenerative disease. The study aims to explore how the TDP43 protein, which accumulates abnormally in ALS, can be effectively degraded by lysosomal proteases. By examining the role of lysosomes in clearing protein aggregates, the research seeks to identify potential therapeutic strategies that could prevent motor neuron death and preserve function in affected individuals. Patients may benefit from insights that could lead to new treatments for ALS.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Amyotrophic Lateral Sclerosis.
Not a fit: Patients with other neurodegenerative disorders unrelated to ALS may not receive benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to novel therapies that slow down or prevent the progression of ALS.
How similar studies have performed: Previous research has shown promise in targeting lysosomal pathways for neurodegenerative diseases, suggesting potential for success in this approach.
Where this research is happening
San Francisco, United States
- University of California, San Francisco — San Francisco, United States (Active)
Researchers
- Principal investigator: Sampognaro, Paul Joseph — University of California, San Francisco
- Study coordinator: Sampognaro, Paul Joseph
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.