Helping cells clear harmful misfolded proteins
Molecular Chaperones and Small Molecules
This project develops small molecules that help cells remove harmful misfolded proteins for people with neurodegenerative conditions like SBMA and frontotemporal dementia.
Quick facts
| Grant type | R01 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-11142430 on NIH RePORTER |
What this research studies
Researchers are developing small molecules that change how a cell 'helper' protein called Hsp70 decides whether to repair or destroy damaged proteins. They use biochemical tools and engineered probes that block Hsp70's interactions with its co-chaperones to see how those changes trigger clearance of toxic proteins such as mutant androgen receptor and tau. The team runs detailed binding and calorimetry experiments and protein-degradation assays in lab models, and has shared reagents with many labs worldwide. The long-term aim is to turn these lab findings into drug targets that could be tested in people with diseases linked to protein misfolding.
Who could benefit from this research
Good fit: Ideal candidates for future trials would be adults diagnosed with spinobulbar muscular atrophy (SBMA), frontotemporal dementia (FTD), or related conditions tied to toxic protein accumulation.
Not a fit: People whose neurologic problems are not driven by protein misfolding, or those with very advanced disease, may be less likely to benefit from these approaches.
Why it matters
Potential benefit: If successful, this work could lead to drugs that restore cells' protein-cleanup systems and slow or prevent progression of diseases like SBMA and FTD.
How similar studies have performed: Laboratory studies have shown that modulating Hsp70 interactions can promote removal of toxic proteins, but benefit in patients has not yet been demonstrated.
Where this research is happening
San Francisco, United States
- University of California, San Francisco — San Francisco, United States (Active)
Researchers
- Principal investigator: Gestwicki, Jason E — University of California, San Francisco
- Study coordinator: Gestwicki, Jason E
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.