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Mouse models available for Polycystic Kidney Disease (PKD) drug testing

Scientists at Leiden University Medical Center have developed mouse models for Polycystic Kidney Disease (PKD) drug testing.

Autosomal Dominant Polycystic Kidney Disease (ADPKD) is a common genetic disease characterized by progressive development of fluid-filled cysts in both kidneys. The formation of numerous cysts together with interstitial fibrosis usually causes chronic renal failure in 50% of patients by the age of 60 years. ADPKD is a systemic disorder that is caused by mutations in the PKD1 or PKD2 genes. The majority of patients, 85%, carry a mutation in the PKD1 gene.

The first mouse model is a tamoxifen-inducible, kidney epithelium-specific Pkd1-deletion model, which shows that inactivation of the Pkd1 gene induces rapid cyst formation in developing kidneys. By selectively inactivating the Pkd1 gene, the embryonic or postnatal lethality is overcome, which makes this model very useful in studying the onset of the disease. A second mouse model has been developed with reduced Pkd1-gene expression. This model is extremely interesting for mimicking the progression and end stages of the disease. Overall, the combination of both mouse models, not hampered by early death, are highly suitable for studying the progression of the disease in vivo and for testing the effect of therapeutic interventions in the different PKD disease stages.

We are interested in speaking with companies with an interest in kidney diseases with a view to a collaboration in this field.

Key benefits

  • The mouse models are based on the genetic defects seen in PKD patients
  • Opportunity to analyze the different stages of the disease


  • Drug testing model for PKD and kidney fibrosis
  • Long-term studies on the pathogenesis of PKD

Development stage

Validated mouse model available 

Luris reference number


Data available on request

Non-confidential and confidential presentations

Further information

Frits Fallaux Knowledge Broker (LU) +31-71-527 2517