Kidney Organoid Derived from Human Pluripotent and Adult Stem Cells for Disease Modeling

Hyun Mi Kang,1,2
Author Information & Copyright
1Korea Research Institute of Bioscience and Biotechnology (KRIBB), Daejeon 34141, Korea
2Department of Functional Genomics, Korea University of Science and Technology (UST), Daejeon 34113, Korea
Corresponding author Hyun Mi Kang, Korea Research Institute of Bioscience and Biotechnology (KRIBB), Daejeon 34141, Korea, Tel: +82-42-879-8173, Fax: +82-42-860-4027, E-mail:

© Copyright 2023 The Korean Society of Developmental Biology. This is an Open-Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License ( which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.

Received: Feb 28, 2023 ; Revised: Apr 27, 2023 ; Accepted: May 26, 2023

Published Online: Jun 30, 2023


Kidney disease affects a significant portion of the global population, yet effective therapies are lacking despite advancements in identifying genetic causes. This limitation can be attributed to the absence of adequate in vitro models that accurately mimic human kidney disease, hindering targeted therapeutic development. However, the emergence of human induced pluripotent stem cells (PSCs) and the development of organoids using them have opened up a way to model kidney development and disease in humans, as well as validate the effects of new drugs. To fully leverage their capabilities in these fields, it is crucial for kidney organoids to closely resemble the structure and functionality of adult human kidneys. In this review, we aim to discuss the potential of using human PSCs or adult kidney stem cell-derived kidney organoids to model genetic kidney disease and renal cancer.

Keywords: Kidney organoid; Genetic disease; Disease modeling


Organoid mimics in vivo organs both structurally and functionally, which enables researchers to gain a deeper understanding of human organ development, physiology, and pathology. Two methods for generating organoids are currently used. The first involves using tissue stem/progenitor cells. In 2009, Hans Clevers and his team showed that leucine-rich repeat-containing G protein-coupled receptor-5 (LGR-5) positive stem cells could generate three-dimensional intestinal organoids (Sato et al., 2009). Since then, many other organoids, such as those of the liver, kidney, pancreas, stomach, uterus, prostate, and mammary gland, have been generated using stem cells from their own respective tissues (Barker et al., 2010; Karthaus et al., 2014; Boj et al., 2015; Hu et al., 2018; Sachs et al., 2018; Fitzgerald et al., 2019; Schutgens et al., 2019). The second method involves using pluripotent stem cells (PSCs), including embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs), which have the capacity for self-renewal and differentiation (Fatehullah et al., 2016). PSCs can differentiate into three germ layer cells using signaling pathways such as wnt, retinoic acid, fibroblast growth factor, bone morphogenic protein, and transforming growth factor (Clevers, 2016). Recently, organoids of various organs, such as the intestine, liver, lung, thyroid, pancreas, brain, retina, and kidney, have been generated using PSCs (Eiraku et al., 2008; Kurmann et al., 2015; Ogawa et al., 2015; Takasato et al., 2015; Chen et al., 2017; Hohwieler et al., 2017; Tsai et al., 2017; Capowski et al., 2019).

Kidney is complex organ that is compose of various different types of cells and it is responsible for many functions, such as filtering and removing the waste products form the body, removing drugs from the body, balancing the fluid in the body, releasing hormones that regulate blood pressure, and control the production red blood cells. The basic functional unit of kidney is called nephron consists of glomerulus, a complex of blood vessel capillaries and podocytes responsible for the filtration of blood, and multiple segment of tubule epithelium responsible for reabsorption and hormone secretion. Loss of functional nephrons and the development of tubulointerstitial fibrosis contribute to the progression of chronic kidney disease, and it affects around 15% of the population and it ultimately leads to end-stage kidney disease (ESKD; Coresh et al., 2007; Eneanya et al., 2016). The patients of ESKD require renal replacement therapies, such as hemodialysis, associated with high morbidity and mortality. Moreover, in vivo mimic disease models for biomarker identification and development of therapeutic approaches for most kidney diseases are limited. A better understanding the basic mechanism of ESKD will be useful to develop novel therapeutic approaches and prevent the disease progression. In general, the causes of kidney failure could be divided into genetic and non-genetic causes. Several differentiation protocols for kidney organoids have been reported from PSCs, and some of these reports mimicked disease models using kidney organoid by genetic mutant line generation. Here, in this review, we discuss genetic kidney disease modeling using PSCs and the kidney cancer organoids using patient-derived samples.


1. Tubular kidney disease

Human PSCs are highly adaptable to genetic manipulation, they can cause genetic abnormalities associated with hereditary kidney disease. Polycystic kidney disease (PKD) has been most frequently studied using PSCs derived kidney organoid. Autosomal dominant polycystic kidney disease (ADPKD) is the most common hereditary kidney disease, and the main feature of ADPKD are multiple renal cysts that eventually cause renal failure, often accompanied by liver, pancreatic cysts, and cerebral aneurysms (Fig. 1A; Torres et al., 2007). PKD1 and PKD2 are the main causative genes for ADPKD, the former accounting for about 85% of cases. CRISPR-Cas9 PKD1-edited biallelic mutant ESCs derived organoid showed formation of cyst-like structures in the proximal tubule of organoid (Freedman et al., 2015; Cruz et al., 2017) and the kidney organoid from CRISPR-Cas9 PKD1 edited human iPSCs cell line exhibited cysts formation in both nephron progenitor and UB/collecting duct tubules in organoid after cyclic adenosin monophosphate (cAMP) stimulation (Kuraoka et al., 2020; Shimizu et al., 2020). Biallelic gene-edited mutant organoids displayed cyst formation following cAMP activation, suggesting that cAMP signaling is important in PKD cystogenesis in cellular models as well as in vivo (Harris & Torres, 2014). Autosomal recessive PKD is characterized by enlarged kidneys with 2 waves of cytogenesis. In ARPKD kidney, proximal tubule cysts are prominent in fetal kidney, and cyst formation dominates in the collecting duct after birth (Nakanishi et al., 2000). Low and colleagues reported the kidney organoid from patient derived biallelic PKHD1 mutant hiPSCs showed cysts formation in the proximal tubules following cAMP stimulation, and CRISPR-Cas9 corrected isogenic lines produce organoids with ameliorated cytogenesis (Low et al., 2019).

Fig. 1. Genetic tubular and glomerular disease. Schematic diagram showing information about genetic kidney diseases and the associated genes. (A) Tubular renal disease and its associated genes. (B) Glomerular renal disease and its associated genes.
Download Original Figure

HNF1B is expressed in developing mouse ureters and collecting ducts, and it is expressed in proximal and distal tubules after birth (Coffinier et al., 1999). HNF1B is expressed in both maturing human collecting ducts and nephrons. Hnf1b deletion in mouse collecting ducts causes cysts and upregulates transcription of uromodulin (Umod), Pkhd1, and Pkd2 (Gresh et al., 2004), and similar results are also observed in human kidneys such as fetal bilateral hyperechogenic kidneys, multi-cystic dysplastic kidneys, and hyperuricemic nephropathy (Fig. 1A; Bingham et al., 2003; Ulinski et al., 2006; Decramer et al., 2007). CRISPR-Cas9 HNF1B edited biallelic or heterozygous mutant iPSCs derived kidney organoid exhibited the reduced proximal tubule, thick ascending limb markers and reduced UB-derived tubule branching (Przepiorski et al., 2018; Mae et al., 2020). hPSCs derived kidney organoids have also been used to model for genetic tubulopathies, such as cystinosin (CTNS) and Mucin-1 (MUC1) mutation for nephropathic cystinosis and medullary cystic kidney disease, respectively (Fig. 1A). In the absence of CTNS, cystine accumulates within the lysosome, causing lysosomal dysfunction. Nephropathy is the most severe form of cystinosis and initially involves the inability of the renal proximal tubules to reabsorb essential metabolites from the urine (Gahl et al., 2002). Hollywood and colleagues generated CTNS mutant iPSCs and showed that kidney organoid from these iPSCs exhibit elevated cystine levels, enlarged lysosomes, increased apoptosis, and defective basal autophagy, and they demonstrated that combined treatment with cysteamine and everolimus, an mTOR pathway inhibitor, rescued the disease phenotype in the mutant hiPSCs derived organoid (Hollywood et al., 2020).

Autosomal dominant tubulo-interstitial kidney disease-mucin1 is caused by a frame-shifting mutation in the GC-rich variable number of tandem repeats region of the MUC1 gene (Kirby et al., 2013). In the kidney, MUC1 localizes to distal convoluted tubules and collecting duct, and after ischemia induction, the protein may be induced in the proximal tubule (Al-Bataineh et al., 2016; Gibier et al., 2017). Missense MUC1 mutation generates a protein accumulation with in the epithelial cells in the patient derived hiPSC kidney organoids leading to damages the tubules by accumulating in transmembrane emp24 domain-containing protein (TMED9) enriched vesicles, and treatment with the small molecule BRD4780 binding cargo receptor TMED9 significantly reduced levels of misfolded MUC1 (Dvela-Levitt et al., 2019).

2. Glomerular kidney disease

Our understanding of glomerular nephropathy is hampered by the limited proliferative nature and architecturally complex structure of the primary podocytes (Lasagni et al., 2013). In the glomerulus, podocytes play an important role in the filtration process and have multiple cytoplasmic protrusions called foot processes. The podocyte foot processes form a specialized cell–cell contact, the slit diaphragm (SD) to prevent leakage of high molecular weight serum proteins into the urine (Schell et al., 2014; Fig. 1B). The main components of SD are NEPHRIN, PODOCIN, and NEPH1. NEPHRIN and HEPH1 are transmembrane proteins that intercalate with proteins from adjacent cells to form a molecular mesh, SD. PODOCIN is thought to stabilize SD by binding to the cytoplasmic region of NEPHRIN (Sharmin et al., 2016). Thus, mutations in these genes cause proteinuria in humans and/or mice. NPHS1 mutated hPSCs derived kidney organoid models reduced levels of nephrin and podocin SD proteins and NPHS1 protein does not localize to the surface of podocyte-like cells (Tanigawa et al., 2018). Mutation in NPHS1 showed abnormality in the SD formation. Podocyte derived from NPHS1 mutant patient iPSCs showed significantly reduced cell surface localization of NEPHRIN despite having normal foot process (Tanigawa et al., 2018). Other study demonstrated that mutation of NPHS1 showed reduced levels of NEPHRIN and PODOCIN SD proteins and large hypertrophied podocyte bodies in the iPSCs derived kidney organoid (Hale et al., 2018). These models are useful for studying of congenital nephrotic syndrome.

Homozygous loss of PODOCALYXIN (PODXL) leads to perinatal retention of junctional complexes between immature podocytes, a walling off the urinary space, renal failure and ultimately, perinatal death (Doyonnas et al., 2001; Kang et al., 2017). Therefore, PODXL mutant hPSCs derive kidney organoid is useful for studying human glomerular development. CRISPR-Cas9 edited biallelic mutant hESCs derived kidney organoid exhibited a lack of microvilli on apical and lateral podocyte cell membrane and reduced lateral spacing (Kim et al., 2017). Moreover, this model showed defective junctional organization and decreased gaps between adjacent podocytes similar to that observed in Podxl null mice (Freedman et al., 2015). This kidney organoids suggest the potential of kidney organoids as an effective model for studying pathological mechanisms for glomerular kidney disease.

3. Renal cancer model

Renal cell carcinomas (RCCs) refer to a group of cancers that can be identified by their distinct genetic mutations. There are about 372,000 new cases of kidney cancer diagnosed worldwide each year, with approximately 166,000 deaths reported in 2019. The three primary subtypes of RCC are clear cell RCC (ccRCC), papillary RCC (pRCC), and chromophobe RCC (chRCC), which account for 75%, 15%, and 5% of RCCs, respectively (Creighton et al., 2013; Davis et al., 2014; Marston Linehan et al., 2016; Hsieh et al., 2017). ccRCC is the most common subtype of RCC and is characterized by a loss of function mutation in the von Hippel-Lindau gene, leading to the accumulation of hypoxia-inducible factor (HIF) and subsequent activation of angiogenesis and cell proliferation pathways. pRCC is characterized by a mutation in the MET gene or its downstream signaling pathways, leading to aberrant cell proliferation and invasion. chRCC is characterized by alterations in mitochondrial genes and metabolic pathways. Inherited mutations can increase the risk of developing RCC and pose a significant challenge, as there is currently a lack of three-dimensional in vitro models for studying cancer progression.

Recent advancements in the field of renal cancer research have led to the development and characterization of tumoroids derived from renal cancer cells. These tumoroids have proven to be effective in preserving critical genetic and phenotypic features of the original tumor tissues. ccRCC-derived tumoroids have exhibited the presence of both epithelial and mesenchymal cells, expressing specific markers associated with renal cancer, such as HIF1α. Importantly, these tumoroids have demonstrated the ability to proliferate even after being transplanted into xenograft models (Grassi et al., 2019). In a separate study conducted a biobank was established using various childhood kidney cancers, including Wilms tumors, RCC, and malignant rhabdoid tumors of the kidney (MRTK). The tumoroids derived from these samples exhibited tri-phasic histology, consisting of epithelial, stromal, and blastema components. Notably, the MRTK tumoroids represented a significant breakthrough as they were the first cancer organoids capable of long-term in vitro expansion for non-epithelial origin tumors (Calandrini et al., 2020). Huang group developed the kidney organoid culture system utilized to generate 33 kidney cancer organoid lines from common subtypes of kidney cancer, including ccRCC, pRCC, and chRCC. These RCC organoids retained the histological structures, mutational characteristics, and transcriptional profiles of the original tumor tissues. Furthermore, single-cell RNA sequencing demonstrated inter- and intra-tumoral heterogeneity in RCC organoids. RCC organoids also enabled in vitro drug screening and offered a means of evaluating the effectiveness of chimeric antigen receptor T cells (Li et al., 2022).

There is a study to report the in vitro model of c-met-mutated hereditary kidney cancer using hiPSCs derived from a patient with type 1 pRCC. The hiPSCs were able to differentiate into 3D kidney organoids that exhibited features of glomeruli, proximal tubules, and expressed markers of pRCC, renal progenitors, and endothelial cells. These organoids were then transplanted under the kidney capsule of NSG mice, where they formed larger tumors compared to the controls. The researchers also found that the gene expression signature of these organoids was highly associated with the expression pattern found in a large cohort of pRCC patient samples. They identified 11 common genes, including BHLHE40 and KDM4C, which are factors involved in pRCC pathogenesis. This study provides a promising in vitro model for studying c-met-mutated hereditary kidney cancer and may lead to better understanding of the disease and development of targeted therapies (Hwang et al., 2019). In addition, Schutgens et al. (2019) used adult stem cells isolated from the urine of patients with various kidney diseases to generate kidney tubuloids. These tubuloids were used to study the effects of BK virus on nephrotic injury, investigate the effects of agents on cystic fibrosis phenotypes, and establish tubuloids from Wilms tumor tissue to study the disease’s pathogenesis (Schutgens et al., 2019). Despite these promising developments, it is important to note that research utilizing renal cancer-derived tumoroids is still in its early stages. Further improvements in the methodology are necessary to enhance the potential applications of these tumoroids in downstream studies and clinical applications. Nevertheless, a lot of studies are currently underway to better understand the molecular mechanisms of RCC and to develop more effective treatments. The use of kidney organoids derived from patient-specific tissue or iPSCs holds promise for personalized drug screening and precision medicine approaches in the treatment of RCC development.


The development of kidney organoids using human adult or PSCs holds great significance in understanding kidney development and modeling kidney diseases. Kidney organoids allow for personalized studies of genetic kidney diseases and drug screening on a human-derived platform. Although they have tremendous promise, kidney organoids currently have limitations in terms of maturity and inability to represent all types of kidney cell. To develop the in vivo mimic human kidney and related diseases, we need to mature them by recapitulating developing kidney interactions, especially between the UB and MM, and focus on the development of vascularized organoids, given the intimate relationship between the kidney and vasculature. Kidney organoids have the potential to be used for genetic studies of adult and fetal kidney disease, drug screening, and personalized medicine development. The progress in kidney organoid technology has the potential to expedite the development of more precise and efficient kidney disease treatments.

Conflict of interests

The author declare no potential conflict of interest.


This research was supported by the Korea Research Institute of Bioscience and Biotechnology (KRIBB) Research Initiative Program (KGM4722122), and the National Research Foundation of Korea funded by the Ministry of Science and ICT (NRF-2019R1C1C1006822).

Authors’ contributions

The article is prepared by a single author.

Ethics approval

This manuscript does not require IRB/IACUC approval because there are no human and animal participants.



Al-Bataineh MM, Kinlough CL, Poland PA, Pastor-Soler NM, Sutton TA, Mang HE, Bastacky SI, Gendler SJ, Madsen CS, Singh S, Monga SP, Hughey RP. 2016; Muc1 enhances the β-catenin protective pathway during ischemia-reperfusion injury. Am J Physiol Renal Physiol. 310:F569-F579


Barker N, Huch M, Kujala P, van de Wetering M, Snippert HJ, van Es JH, Sato T, Stange DE, Begthel H, van den Born M, Danenberg E, van den Brink S, Korving J, Abo A, Peters PJ, Wright N, Poulsom R, Clevers H. 2010; Lgr5+ve stem cells drive self-renewal in the stomach and build long-lived gastric units in vitro. Cell Stem Cell. 6:25-36


Bingham C, Ellard S, Van’t Hoff WG, Anne Simmonds H, Marinaki AM, Badman MK, Winocour PH, Stride A, Lockwood CR, Nicholls AJ, Owen KR, Spyer G, Pearson ER, Hattersley AT. 2003; Atypical familial juvenile hyperuricemic nephropathy associated with a hepatocyte nuclear factor-1β gene mutation. Kidney Int. 63:1645-1651


Boj SF, Hwang CI, Baker LA, Chio IIC, Engle DD, Corbo V, et al. 2015; Organoid models of human and mouse ductal pancreatic cancer. Cell. 160:324-338


Calandrini C, Schutgens F, Oka R, Margaritis T, Candelli T, Mathijsen L, et al. 2020; An organoid biobank for childhood kidney cancers that captures disease and tissue heterogeneity. Nat Commun. 11:1310


Capowski EE, Samimi K, Mayerl SJ, Joseph Phillips M, Pinilla I, Howden SE, Saha J, Jansen AD, Edwards KL, Jager LD, Barlow K, Valiauga R, Erlichman Z, Hagstrom A, Sinha D, Sluch VM, Chamling X, Zack DJ, Skala MC, Gamm DM. 2019; Reproducibility and staging of 3D human retinal organoids across multiple pluripotent stem cell lines. Development. 146:dev171686


Chen YW, Huang SX, de Carvalho ALRT, Ho SH, Islam MN, Volpi S, Notarangelo LD, Ciancanelli M, Casanova JL, Bhattacharya J, Liang AF, Palermo LM, Porotto M, Moscona A, Snoeck HW. 2017; A three-dimensional model of human lung development and disease from pluripotent stem cells. Nat Cell Biol. 19:542-549


Clevers H. 2016; Modeling development and disease with organoids. Cell. 165:1586-1597


Coffinier C, Barra J, Babinet C, Yaniv M. 1999; Expression of the vHNF1/HNF1β homeoprotein gene during mouse organogenesis. Mech Dev. 89:211-213


Coresh J, Selvin E, Stevens LA, Manzi J, Kusek JW, Eggers P, Van Lente F, Levey AS. 2007; Prevalence of chronic kidney disease in the United States. J Am Med Assoc. 298:2038-2047


Creighton CJ, Morgan M, Gunaratne PH, Wheeler DA, Gibbs RA, Robertson AG, et al. 2013; Comprehensive molecular characterization of clear cell renal cell carcinoma. Nature. 499:43-49


Cruz NM, Song X, Czerniecki SM, Gulieva RE, Churchill AJ, Kim YK, Winston K, Tran LM, Diaz MA, Fu H, Finn LS, Pei Y, Himmelfarb J, Freedman BS. 2017; Organoid cystogenesis reveals a critical role of microenvironment in human polycystic kidney disease. Nat Mater. 16:1112-1119


Davis CF, Ricketts CJ, Wang M, Yang L, Cherniack AD, Shen H, et al. 2014; The somatic genomic landscape of chromophobe renal cell carcinoma. Cancer Cell. 26:319-330


Decramer S, Parant O, Beaufils S, Clauin S, Guillou C, Kessler S, Aziza J, Bandin F, Schanstra JP, Bellanné-Chantelot C. 2007; Anomalies of the TCF2 gene are the main cause of fetal bilateral hyperechogenic kidneys. J Am Soc Nephrol. 18:923-933


Doyonnas R, Kershaw DB, Duhme C, Merkens H, Chelliah S, Graf T, McNagny KM. 2001; Anuria, omphalocele, and perinatal lethality in mice lacking the CD34-related protein podocalyxin. J Exp Med. 194:13-28


Dvela-Levitt M, Kost-Alimova M, Emani M, Kohnert E, Thompson R, Sidhom EH, et al. 2019; Small molecule targets TMED9 and promotes lysosomal degradation to reverse proteinopathy. Cell. 178:521-535.E23


Eiraku M, Watanabe K, Matsuo-Takasaki M, Kawada M, Yonemura S, Matsumura M, Wataya T, Nishiyama A, Muguruma K, Sasai Y. 2008; Self-organized formation of polarized cortical tissues from ESCs and its active manipulation by extrinsic signals. Cell Stem Cell. 3:519-532


Eneanya ND, Wenger JB, Waite K, Crittenden S, Hazar DB, Volandes A, Temel JS, Thadhani R, Paasche-Orlow MK. 2016; Racial disparities in end-of-life communication and preferences among chronic kidney disease patients. Am J Nephrol. 44:46-53


Fatehullah A, Tan SH, Barker N. 2016; Organoids as an in vitro model of human development and disease. Nat Cell Biol. 18:246-254


Fitzgerald HC, Dhakal P, Behura SK, Schust DJ, Spencer TE. 2019; Self-renewing endometrial epithelial organoids of the human uterus. Proc Natl Acad Sci USA. 116:23132-23142


Freedman BS, Brooks CR, Lam AQ, Fu H, Morizane R, Agrawal V, et al. 2015; Modelling kidney disease with CRISPR-mutant kidney organoids derived from human pluripotent epiblast spheroids. Nat Commun. 6:8715


Gahl WA, Thoene JG, Schneider JA. 2002; Cystinosis. N Engl J Med. 347:111-121


Gibier JB, Hémon B, Fanchon M, Gaudelot K, Pottier N, Ringot B, Van Seuningen I, Glowacki F, Cauffiez C, Blum D, Copin MC, Perrais M, Gnemmi V. 2017; Dual role of MUC1 mucin in kidney ischemia-reperfusion injury: Nephroprotector in early phase, but pro-fibrotic in late phase. Biochim Biophys Acta Mol Basis Dis. 1863:1336-1349


Grassi L, Alfonsi R, Francescangeli F, Signore M, De Angelis ML, Addario A, et al. 2019; Organoids as a new model for improving regenerative medicine and cancer personalized therapy in renal diseases. Cell Death Dis. 10:201


Gresh L, Fischer E, Reimann A, Tanguy M, Garbay S, Shao X, Hiesberger T, Fiette L, Igarashi P, Yaniv M, Pontoglio M. 2004; A transcriptional network in polycystic kidney disease. EMBO J. 23:1657-1668


Hale LJ, Howden SE, Phipson B, Lonsdale A, Er PX, Ghobrial I, Hosawi S, Wilson S, Lawlor KT, Khan S, Oshlack A, Quinlan C, Lennon R, Little MH. 2018; 3D organoid-derived human glomeruli for personalised podocyte disease modelling and drug screening. Nat Commun. 9:5167


Harris PC, Torres VE. 2014; Genetic mechanisms and signaling pathways in autosomal dominant polycystic kidney disease. J Clin Invest. 124:2315-2324


Hohwieler M, Illing A, Hermann PC, Mayer T, Stockmann M, Perkhofer L, et al. 2017; Human pluripotent stem cell-derived acinar/ductal organoids generate human pancreas upon orthotopic transplantation and allow disease modelling. Gut. 66:473-486


Hollywood JA, Przepiorski A, D’souza RF, Sreebhavan S, Wolvetang EJ, Harrison PT, Davidson AJ, Holm TM. 2020; Use of human induced pluripotent stem cells and kidney organoids to develop a cysteamine/mTOR inhibition combination therapy for cystinosis. J Am Soc Nephrol. 31:962-982


Hsieh JJ, Purdue MP, Signoretti S, Swanton C, Albiges L, Schmidinger M, Heng DY, Larkin J, Ficarra V. 2017; Renal cell carcinoma. Nat Rev Dis Primers. 3:17009


Hu H, Gehart H, Artegiani B, Löpez-Iglesias C, Dekkers F, Basak O, et al. 2018; Long-term expansion of functional mouse and human hepatocytes as 3D organoids. Cell. 175:1591-1606.E19


Hwang JW, Desterke C, Féraud O, Richard S, Ferlicot S, Verkarre V, Patard JJ, Loisel-Duwattez J, Foudi A, Griscelli F, Bennaceur-Griscelli A, Turhan AG. 2019; iPSC-derived embryoid bodies as models of c-Met-mutated hereditary papillary renal cell carcinoma. Int J Mol Sci. 20:4867


Kang HG, Lee M, Lee KB, Hughes M, Kwon BS, Lee S, McNagny KM, Ahn YH, Ko JM, Ha IS, Choi M, Cheong HI. 2017; Loss of podocalyxin causes a novel syndromic type of congenital nephrotic syndrome. Exp Mol Med. 49:e414


Karthaus WR, Iaquinta PJ, Drost J, Gracanin A, van Boxtel R, Wongvipat J, Dowling CM, Gao D, Begthel H, Sachs N, Vries RGJ, Cuppen E, Chen Y, Sawyers CL, Clevers HC. 2014; Identification of multipotent luminal progenitor cells in human prostate organoid cultures. Cell. 159:163-175


Kim YK, Refaeli I, Brooks CR, Jing P, Gulieva RE, Hughes MR, Cruz NM, Liu Y, Churchill AJ, Wang Y, Fu H, Pippin JW, Lin LY, Shankland SJ, Wayne Vogl A, McNagny KM, Freedman BS. 2017; Gene-edited human kidney organoids reveal mechanisms of disease in podocyte development. Stem Cells. 35:2366-2378


Kirby A, Gnirke A, Jaffe DB, Barešová V, Pochet N, Blumenstiel B, et al. 2013; Mutations causing medullary cystic kidney disease type 1 lie in a large VNTR in MUC1 missed by massively parallel sequencing. Nat Genet. 45:299-303


Kuraoka S, Tanigawa S, Taguchi A, Hotta A, Nakazato H, Osafune K, Kobayashi A, Nishinakamura R. 2020; PKD1-dependent renal cystogenesis in human induced pluripotent stem cell-derived ureteric bud/collecting duct organoids. J Am Soc Nephrol. 31:2355-2371


Kurmann AA, Serra M, Hawkins F, Rankin SA, Mori M, Astapova I, Ullas S, Lin S, Bilodeau M, Rossant J, Jean JC, Ikonomou L, Deterding RR, Shannon JM, Zorn AM, Hollenberg AN, Kotton DN. 2015; Regeneration of thyroid function by transplantation of differentiated pluripotent stem cells. Cell Stem Cell. 17:527-542


Lasagni L, Lazzeri E, Shankland SJ, Anders HJ, Romagnani P. 2013; Podocyte mitosis: A catastrophe. Curr Mol Med. 13:13-23


Li Z, Xu H, Yu L, Wang J, Meng Q, Mei H, Cai Z, Chen W, Huang W. 2022; Patient-derived renal cell carcinoma organoids for personalized cancer therapy. Clin Transl Med. 12:e970


Low JH, Li P, Chew EGY, Zhou B, Suzuki K, Zhang T, Lian MM, Liu M, Aizawa E, Rodriguez Esteban C, Yong KSM, Chen Q, Campistol JM, Fang M, Khor CC, Foo JN, Izpisua Belmonte JC, Xia Y. 2019; Generation of human PSC-derived kidney organoids with patterned nephron segments and a de novo vascular network. Cell Stem Cell. 25:373-387.E9


Mae SI, Ryosaka M, Sakamoto S, Matsuse K, Nozaki A, Igami M, Kabai R, Watanabe A, Osafune K. 2020; Expansion of human iPSC-derived ureteric bud organoids with repeated branching potential. Cell Rep. 32:107963


Marston Linehan W, Spellman PT, Ricketts CJ, Creighton CJ, Fei SS, Davis C, et al. 2016; Comprehensive molecular characterization of papillary renal-cell carcinoma. N Engl J Med. 374:135-145


Nakanishi K, Sweeney WE, Zerres K, Guay-Woodford LM, Avner ED. 2000; Proximal tubular cysts in fetal human autosomal recessive polycystic kidney disease. J Am Soc Nephrol. 11:760-763


Ogawa M, Ogawa S, Bear CE, Ahmadi S, Chin S, Li B, Grompe M, Keller G, Kamath BM, Ghanekar A. 2015; Directed differentiation of cholangiocytes from human pluripotent stem cells. Nat Biotechnol. 33:853-861


Przepiorski A, Sander V, Tran T, Hollywood JA, Sorrenson B, Shih JH, Wolvetang EJ, McMahon AP, Holm TM, Davidson AJ. 2018; A simple bioreactor-based method to generate kidney organoids from pluripotent stem cells. Stem Cell Rep. 11:470-484


Sachs N, de Ligt J, Kopper O, Gogola E, Bounova G, Weeber F, et al. 2018; A living biobank of breast cancer organoids captures disease heterogeneity. Cell. 172:373-386.E10


Sato T, Vries RG, Snippert HJ, van de Wetering M, Barker N, Stange DE, van Es JH, Abo A, Kujala P, Peters PJ, Clevers H. 2009; Single Lgr5 stem cells build crypt-villus structures in vitro without a mesenchymal niche. Nature. 459:262-265


Schell C, Wanner N, Huber TB. 2014; Glomerular development – shaping the multi-cellular filtration unit. Semin Cell Dev Biol. 36:39-49


Schutgens F, Rookmaaker MB, Margaritis T, Rios A, Ammerlaan C, Jansen J, et al. 2019; Tubuloids derived from human adult kidney and urine for personalized disease modeling. Nat Biotechnol. 37:303-313


Sharmin S, Taguchi A, Kaku Y, Yoshimura Y, Ohmori T, Sakuma T, Mukoyama M, Yamamoto T, Kurihara H, Nishinakamura R. 2016; Human induced pluripotent stem cell–derived podocytes mature into vascularized glomeruli upon experimental transplantation. J Am Soc Nephrol. 27:1778-1791


Shimizu T, Mae SI, Araoka T, Okita K, Hotta A, Yamagata K, Osafune K. 2020; A novel ADPKD model using kidney organoids derived from disease-specific human iPSCs. Biochem Biophys Res Commun. 529:1186-1194


Takasato M, Er PX, Chiu HS, Maier B, Baillie GJ, Ferguson C, Parton RG, Wolvetang EJ, Roost MS, Chuva de Sousa Lopes SM, Little MH. 2015; Kidney organoids from human iPS cells contain multiple lineages and model human nephrogenesis. Nature. 536:238


Tanigawa S, Islam M, Sharmin S, Naganuma H, Yoshimura Y, Haque F, Era T, Nakazato H, Nakanishi K, Sakuma T, Yamamoto T, Kurihara H, Taguchi A, Nishinakamura R. 2018; Organoids from nephrotic disease-derived iPSCs identify impaired NEPHRIN localization and slit diaphragm formation in kidney podocytes. Stem Cell Reports. 11:727-740


Torres VE, Harris PC, Pirson Y. 2007; Autosomal dominant polycystic kidney disease. Lancet. 369:1287-1301


Tsai YH, Nattiv R, Dedhia PH, Nagy MS, Chin AM, Thomson M, Klein OD, Spence JR. 2017; In vitro patterning of pluripotent stem cell-derived intestine recapitulates in vivo human development. Development. 144:1045-1055


Ulinski T, Lescure S, Beaufils S, Guigonis V, Decramer S, Morin D, Clauin S, Deschênes G, Bouissou F, Bensman A, Bellanné-Chantelot C. 2006; Renal phenotypes related to hepatocyte nuclear factor-1β (TCF2) mutations in a pediatric cohort. J Am Soc Nephrol. 17:497-503