How polycystin proteins get to and work in tiny cilia of kidney cells
Investigate the trafficking and function of polycystins in primary cilia and their implications for polycystic kidney disease
['FUNDING_R01'] · MAYO CLINIC ROCHESTER · NIH-11307659
Researchers are looking at ways to boost the presence and function of polycystin proteins in tiny antennae of kidney cells to help people with autosomal dominant polycystic kidney disease (ADPKD).
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | MAYO CLINIC ROCHESTER (nih funded) |
| Locations | 1 site (ROCHESTER, UNITED STATES) |
| Trial ID | NIH-11307659 on ClinicalTrials.gov |
What this research studies
This project focuses on how polycystin proteins are transported to and operate within primary cilia, the small sensory structures on kidney cells, using molecular lab experiments, advanced imaging, and genetic models. The team will study three specific pathways that control polycystin delivery and signaling at the ciliary base, entry into the cilium, and cAMP/PKA signaling when polycystins are missing. Experiments will use cell systems and animal models to see how changing these pathways affects cyst formation and kidney cell behavior. Results are intended to identify targets or strategies to restore ciliary polycystin function and reduce cyst growth in ADPKD.
Who could benefit from this research
Good fit: Adults with autosomal dominant polycystic kidney disease (ADPKD), particularly those with PKD1 or PKD2 mutations or early-stage cystic disease, would be most relevant to the aims of this research.
Not a fit: People without ADPKD or with kidney disease from unrelated causes are unlikely to receive direct benefit from this specific line of research.
Why it matters
Potential benefit: If successful, this work could point to new ways to restore polycystin function in kidney cells and lead to treatments that slow or prevent cyst growth in ADPKD.
How similar studies have performed: Previous studies in models have shown that restoring polycystin levels or modulating cAMP signaling can slow cyst growth, but the detailed trafficking mechanisms targeted here are relatively new and less tested.
Where this research is happening
ROCHESTER, UNITED STATES
- MAYO CLINIC ROCHESTER — ROCHESTER, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: LING, KUN — MAYO CLINIC ROCHESTER
- Study coordinator: LING, KUN
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.
Conditions: Adult Polycystic Kidney Disease