ABSTRACT
Kidney organoid cultures derived from human pluripotent stem cells have recently emerged as a new system for studying PKD, which bridges the gap between simple cells and animal models. This “kidney in a dish” organoid system has several strengths, including the formation of human cysts from kidney tubules in a PKD-specific way. Here, we briefly review the human kidney organoid system and its components, focusing on the aspects most relevant to PKD. Sources and characteristics of PKD-mutant stem cells, including reprogrammed and gene-edited lines, are described. We then proceed to outline step-by-step protocols for generating kidney organoids and using them to reconstitute PKD cystogenesis. Methodologies for assessing the expression and localization of polycystins in these structures are also detailed. The advantages, limitations, and pitfalls of the system for disease modeling are outlined, to aid the newcomer in adopting these techniques to understand PKD and test therapeutic approaches.
