Molecules that control polycystin signaling in polycystic kidney disease
Molecular modulators of polycystin signaling
This project looks at proteins and genes that change how polycystin signals in people with autosomal dominant polycystic kidney disease (ADPKD) to find new targets for treatment.
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-11297810 on NIH RePORTER |
What this research studies
From my perspective, researchers are mapping a cilia-dependent cyst activation (CDCA) signaling pattern in kidney tubule cells to find genes that drive cyst growth when polycystin proteins fail. They use lab-grown kidney cells, genetic mouse models, and molecular tools including translatome profiling and chromatin assays such as ATAC-seq to pinpoint functional drivers. The team previously found that blocking one protein, Glis2, reduced cyst growth in mice, and now they will test other candidate genes from the CDCA pattern the same way. The aim is to turn those findings into drug targets that could lead to new treatments for people with ADPKD.
Who could benefit from this research
Good fit: This research is most relevant to people with ADPKD, especially those with PKD1 or PKD2 mutations, though the current work focuses on lab and animal studies rather than enrolling patients.
Not a fit: People without ADPKD, and those already on dialysis or who have received a kidney transplant, are unlikely to benefit directly from the experiments funded by this grant.
Why it matters
Potential benefit: If successful, this work could identify new drug targets that slow or stop cyst growth in ADPKD and lead to better treatments.
How similar studies have performed: Related laboratory and mouse studies, including Glis2 inactivation, have shown promising results in slowing cyst growth, but these approaches remain untested in humans.
Where this research is happening
New Haven, United States
- Yale University — New Haven, United States (Active)
Researchers
- Principal investigator: Somlo, Stefan — Yale University
- Study coordinator: Somlo, Stefan
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.