Understanding kidney protection mechanisms in mouse models of a genetic kidney disease.

Decoding reno-protective mechanisms in mouse Pkhd1 models: Implications for ARPKD therapeutics

NIH-funded research Children's Research Institute · NIH-10817867

This study is looking at how certain proteins help protect kidneys in mice with a genetic condition called ARPKD, which causes serious kidney problems, to find out why mice don’t get as sick as people do, and the hope is that this could lead to new treatments for those living with ARPKD.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionChildren's Research Institute NIH-funded
Lab location1 site (Washington, United States)
Project IDNIH-10817867 on NIH RePORTER

What this research studies

This research investigates the mechanisms that protect kidneys in mouse models of autosomal recessive polycystic kidney disease (ARPKD), a condition that leads to severe kidney dysfunction. By studying how mutations in the PKHD1 gene affect kidney function, the researchers aim to uncover why mice with similar mutations do not exhibit the same severe symptoms as humans. The approach involves analyzing the role of a protein called fibrocystin/polyductin and its interactions within kidney cells. Insights gained from these studies could lead to new therapeutic strategies for patients suffering from ARPKD.

Who could benefit from this research

Good fit: Ideal candidates for this research are individuals diagnosed with autosomal recessive polycystic kidney disease (ARPKD).

Not a fit: Patients with other types of kidney diseases unrelated to ARPKD may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could lead to innovative treatments that improve kidney function and quality of life for patients with ARPKD.

How similar studies have performed: Previous research has shown promising results in understanding kidney disease mechanisms through genetic models, suggesting that this approach could yield valuable insights.

Where this research is happening

Washington, United States

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Conditions DisorderDisease
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.