Understanding and targeting the role of lysosomes in neurodegeneration
Official title: Deciphering and disabling lysosome-dependent mechanisms of neurodegeneration
Funded at University of California Berkeley · NIH-11064059 · Data updated 2026-11-12
This study is looking at how certain problems in our cells, especially in a condition called Niemann-Pick type C, affect the way our cells clean up and manage waste, with the hope of finding new treatments that could help people with this and similar neurodegenerative diseases.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California Berkeley NIH-funded |
| Lab location | 1 site (Berkeley, United States) |
| Project start | April 15, 2024 |
| Project end | January 31, 2029 |
| Funding year | 2025 |
| Award (this year) | $518,567 |
| NIH project number | 5R01NS133575-02 |
| Project ID | NIH-11064059 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals diagnosed with Niemann-Pick type C or related neurodegenerative disorders.
✕Less likely to help
Patients with neurodegenerative diseases not related to lysosomal dysfunction may not benefit from this research.
What this research studies
This research investigates how lysosomes, which are essential for clearing damaged cellular components, function in neurodegenerative diseases like Niemann-Pick type C (NPC). The study focuses on the NPC1 gene, whose mutations lead to cholesterol accumulation in lysosomes, causing neuronal cell death. By examining the molecular mechanisms behind lysosomal dysfunction and its effects on mitochondrial health, the researchers aim to develop targeted therapies to restore normal cell function. Patients may benefit from insights that could lead to new treatments for neurodegenerative conditions.
Why it matters
Potential benefit: If successful, this research could lead to innovative therapies that improve neuronal health and slow the progression of neurodegenerative diseases.
How similar studies have gone: Previous research has shown promising results in understanding lysosomal function in neurodegeneration, suggesting that this approach may yield significant insights.
Where this research is happening
-
University of California Berkeley
Berkeley, United States Active
Researchers
- Principal investigator: Zoncu, Roberto — University of California Berkeley
- Study coordinator: Zoncu, Roberto
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.