How a tail piece of a kidney protein may affect polycystic kidney disease
Polycystin-1 C terminal tail cleavage: Mechanisms and meaning
Researchers are looking at whether the end piece of the polycystin-1 protein can slow or stop cyst growth in people with autosomal dominant polycystic kidney disease.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Yale University NIH-funded |
| Lab location | 1 site (New Haven, United States) |
| Project ID | NIH-11249675 on NIH RePORTER |
What this research studies
This project examines how the C-terminal tail of the polycystin-1 (PC1) protein is cut and where the pieces go inside cells, using lab-grown cells and mouse models that mimic human ADPKD. Scientists will focus on a fragment corresponding to the last 200 amino acids of PC1 (P200) that has been linked to reduced cyst growth in mice. The work will track P200’s movement to parts of the cell like mitochondria and the nucleus and test how those movements change cell behavior and cyst formation. Findings are intended to explain the mechanism so future therapies could target the same process.
Who could benefit from this research
Good fit: Adults with autosomal dominant polycystic kidney disease, especially those with PKD1-related disease or who are interested in participating in future translational studies or tissue donation, would be most relevant.
Not a fit: People without ADPKD or those already with end-stage kidney failure unlikely to receive direct benefit from this preclinical lab-focused work in the near term.
Why it matters
Potential benefit: If successful, this could point to new ways to slow cyst growth and preserve kidney function in people with ADPKD.
How similar studies have performed: Prior preclinical work in mouse models showed that expressing the PC1 C-terminal fragment (P200) can suppress cysts, but the exact mechanisms remain unclear and are the focus here.
Where this research is happening
New Haven, United States
- Yale University — New Haven, United States (Active)
Researchers
- Principal investigator: Caplan, Michael J. — Yale University
- Study coordinator: Caplan, Michael J.
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.