Improving the cell's protein-cleanup machines to fight misfolding diseases
Elucidating the roles of protein disaggregases and developing enhanced diaggregases to counter diverse protein-misfolding disorders
The team is building stronger versions of the cell's protein 'cleanup' machines to help people with diseases caused by misfolded proteins, such as some neurodegenerative, heart, kidney conditions, and certain cancers.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Washington University NIH-funded |
| Lab location | 1 site (Saint Louis, United States) |
| Project ID | NIH-11144957 on NIH RePORTER |
What this research studies
This project looks at how cells normally keep proteins folded and what goes wrong when that system fails in disease. Researchers are engineering and testing enhanced protein 'disaggregases' (including modified Hsp104 variants) to dissolve toxic protein clumps and help proteins return to their proper shape. They will perform lab experiments in cells and model systems to understand mechanisms and optimize the engineered proteins. Successful lab results could guide future therapies or clinical studies aimed at clearing harmful protein aggregates in patients.
Who could benefit from this research
Good fit: People with diseases linked to protein misfolding—for example Alzheimer's or Parkinson's disease, certain cardiomyopathies, kidney disorders, and some cancers—are the patient groups this work aims to help.
Not a fit: People whose conditions are not related to protein misfolding or who need immediate, approved clinical care are unlikely to receive direct benefit from this laboratory-focused research.
Why it matters
Potential benefit: If successful, this work could lead to new ways to clear toxic protein clumps and restore cell function in diseases like Alzheimer's, Parkinson's, some heart and kidney disorders, and certain cancers.
How similar studies have performed: Prior laboratory studies have shown engineered disaggregases can break up protein aggregates in cells and animal models, but turning those findings into safe, effective human treatments is still early-stage and experimental.
Where this research is happening
Saint Louis, United States
- Washington University — Saint Louis, United States (Active)
Researchers
- Principal investigator: Jackrel, Meredith E. — Washington University
- Study coordinator: Jackrel, Meredith 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.