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Work in the laboratories of the authors is funded by FEDER funds through the Operational Programme Competitiveness Factors—COMPETE and national funds by FCT—Foundation for Science and Technology (PTDC/BTM-SAL/29297/2017, POCI-01-0145-FEDER-029297, PTDC/MED-FAR/29391/2017, POCI-01-0145-FEDER-029391, IF/01182/2015 and UIDB/04539/2020). iNOVA4Health-UID/Multi/04462/2013, a program financially supported by Fundação para a Ciência e Tecnologia/Ministério da Educação e Ciência, through national funds and co-funded by FEDER under the PT2020 Partnership Agreement, is acknowledged. Guida Bento is the recipient of a Ph.D. fellowship from the Foundation for Science and Technology (PD/BD/114119/2015). AKS and AAR were funded by the subsidy allocated to Kazan Federal University for the state assignment in the sphere of scientific activities. Kazan Federal University was supported by the Russian Government Program of Competitive Growth. Despite being a biological waste, human urine contains a small population of cells with self-renewal capacity and differentiation potential into several cell types. Being derived from the convoluted tubules of nephron, renal pelvis, ureters, bladder and urethra, urine-derived stem cells (UDSC) have a similar phenotype to mesenchymal stroma cells (MSC) and can be reprogrammed into iPSC (induced pluripotent stem cells). Having simple, safer, low-cost and noninvasive collection procedures, the interest in UDSC has been growing in the last decade. With great potential in regenerative medicine applications, UDSC can also be used as biological models for pharmacology and toxicology tests. This review describes UDSC biological characteristics and differentiation potential and their possible use, including the potential of UDSC-derived iPSC to be used in drug discovery and toxicology, as well as in regenerative medicine. Being a new cellular platform amenable to noninvasive collection for disease stratification and personalized therapy could be a future application for UDSC. publishersversion published |