How cilia changes lead to inherited kidney cyst disorders
Nephronophthisis-related ciliopathies and ciliary specialization
['FUNDING_R01'] · RUTGERS, THE STATE UNIV OF N.J. · NIH-11247554
Researchers will look at tiny cell antennae called cilia to learn how changes in them can cause inherited cystic kidney diseases like nephronophthisis so this work may help affected patients in the future.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | RUTGERS, THE STATE UNIV OF N.J. (nih funded) |
| Locations | 1 site (PISCATAWAY, UNITED STATES) |
| Trial ID | NIH-11247554 on ClinicalTrials.gov |
What this research studies
This lab project uses a tiny worm (C. elegans) that has specialized cilia to learn how cilia change shape and shed small particles called extracellular vesicles. Scientists will use genetic tools and high-resolution imaging to track the proteins and structures that control ciliary remodeling and vesicle release. The team will compare those mechanisms to what is known about human kidney cilia to link basic findings to cystic kidney disease. This is laboratory research aimed at identifying molecular steps that could become targets for future therapies rather than a treatment trial.
Who could benefit from this research
Good fit: People with inherited cystic kidney conditions (for example, nephronophthisis or other ciliopathy-related kidney disease) or those with a family history who might be asked to donate samples for related translational studies.
Not a fit: Patients with unrelated kidney problems (such as diabetic kidney disease or kidney stones) are unlikely to benefit directly from this basic lab research.
Why it matters
Potential benefit: If successful, the work could reveal why ciliary defects lead to kidney cysts and point to new targets for drugs or diagnostics for inherited cystic kidney diseases.
How similar studies have performed: Previous basic research in worms and cell systems has shown cilia can shed vesicles and that ciliary genes cause kidney cysts, but directly linking ciliary vesicle biology to human kidney disease remains largely unproven.
Where this research is happening
PISCATAWAY, UNITED STATES
- RUTGERS, THE STATE UNIV OF N.J. — PISCATAWAY, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: BARR, MAUREEN M — RUTGERS, THE STATE UNIV OF N.J.
- Study coordinator: BARR, MAUREEN M
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, Autosomal Dominant Polycystic Kidney Disease, Autosomal Recessive Medullary Cystic Disease