Stopping lysosome-driven brain cell damage
Deciphering and disabling lysosome-dependent mechanisms of neurodegeneration
Researchers are working to stop harmful changes in cell recycling centers (lysosomes) that lead to brain cell loss in people with Niemann‑Pick type C and related conditions such as Alzheimer's.
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 ID | NIH-11260241 on NIH RePORTER |
What this research studies
I’m learning that tiny cell recycling centers called lysosomes must work properly for neurons to survive. The team studies cells with NPC1 gene mutations and uses patient-derived stem cell neurons to see how cholesterol builds up in lysosomes and breaks the cell’s cleanup systems. They isolate and profile lysosomes to measure enzyme content and membrane stability and follow how damaged mitochondria are (or aren’t) delivered to lysosomes during autophagy. By linking lysosomal failure to mitochondrial and metabolic problems, they aim to identify targets that could be corrected to restore healthy neuron function.
Who could benefit from this research
Good fit: Ideal participants would be people affected by Niemann‑Pick type C or patients with lysosome-related neurodegenerative conditions who can donate cells or enroll in future translational studies or follow-up visits.
Not a fit: Patients with unrelated medical problems or neurodegeneration driven by non-lysosomal mechanisms are unlikely to see direct benefits from this laboratory-focused project in the near term.
Why it matters
Potential benefit: If successful, this work could point to new treatments that prevent or slow neuron loss in NPC and other lysosome-related neurodegenerative diseases, including some forms of Alzheimer's.
How similar studies have performed: Previous laboratory and cell-based studies have shown lysosomal faults in NPC and Alzheimer-related models, but turning those findings into effective therapies remains largely experimental.
Where this research is happening
Berkeley, United States
- 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 below.