Autosomal Dominant Polycystic Kidney Disease (ADPKD) is a slowly progressive genetic renal disorder that is caused by mutations in either the PKD1 gene (polycystin 1) or the PKD2 gene (polycystin 2). Autosomal Recessive Polycystic Kidney Disease (ARPKD) is a rare genetic disorder caused by mutations in PKHD1 gene. Both forms of PKD are characterized by the formation of fluid filled cysts in both kidneys leading eventually to end-stage renal disease. Several in vivo and in vitro models of PKD exist, although many of them fall short of fully recapitulating the human disease phenotype, driving the failure of most therapeutic candidates in human clinical trials.
Our Model System with Patient-Derived Human PKD Cells
We have developed a unique 3D PKD Cyst Assay platform in 384-well tissue culture format utilizing cells isolated from individual cysts on human PKD donor kidneys, where germline mutations are determined. Once in culture, these cells form cysts over days to weeks that can be tracked by high-content imaging with cyst size and number quantified through algorithm-based image analysis. This platform can be used for high-throughput screening or validation of candidate drugs, and provides significant advancement in throughput and pathophysiologic relevancy.
We continuously add to our diverse PKD donor cell repository. Please complete a brief form to be in contact with a Translational Biology expert to discuss your needs. You will be contacted within 1-2 business days. Or email us directly: ClientServicesStCharles@discovery.eurofinsus.com
