Experimental Models of Polycystic Kidney Disease: Applications and Therapeutic Testing.

ABSTRACT: Polycystic kidney diseases (PKDs) are genetic disorders characterized by the formation and expansion of numerous fluid-filled renal cysts, damaging normal parenchyma and often leading to kidney failure. Although PKDs comprise a broad range of different diseases, with substantial genetic and phenotypic heterogeneity, an association with primary cilia represents a common theme. Great strides have been made in the identification of causative genes, furthering our understanding of the genetic complexity and disease mechanisms, but only one therapy so far has shown success in clinical trials and advanced to US Food and Drug Administration approval. A key step in understanding disease pathogenesis and testing potential therapeutics is developing orthologous experimental models that accurately re

SUBMITTER: Sieben CJ 

PROVIDER: S-EPMC10476690 | biostudies-literature | 2023 Aug

REPOSITORIES: biostudies-literature