Urine-derived Human Renal Progenitor Cultures For Modeling Of Genetic Kidney Disorders

In this study, we describe a method to select and amplify RPC cultures from the urine of patients with kidney disorders. U-RPC exhibited identical phenotype and functional properties to those purified from kidney tissue, including the capacity to differentiate into tubular cells as well as podocytes. To evaluate the possibility to use these cells for modeling of genetic kidney disorders, u-RPC were obtained from children affected by nephrotic syndrome carrying putative pathogenic mutations in genes encoding for podocyte cytoskeleton proteins, as well as from children without genetic alterations. U-RPC obtained from patients carrying pathogenic mutations generated podocytes that exhibited altered synthesis of mutated proteins, abnormal cytoskeleton structure and functional abnormalities. By contrast, podocytes differentiated from u-RPC obtained from patients without genetic mutations showed normal phenotype, structure and function.