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Review Open Access

Kidney organoids as models for hereditary kidney diseases: Toward precision medicine (Review)

  • Authors:
    • Sheng Cui
    • Tianlei Chen
    • Yun Zou
    • Min Li
    • Hua Zhou
    • Jingting Jiang
    • Min Yang
  • View Affiliations / Copyright

    Affiliations: Department of Nephrology, The Third Affiliated Hospital of Soochow University, Changzhou, Jiangsu 213003, P.R. China, Department of Tumor Biological Treatment, The Third Affiliated Hospital of Soochow University, Changzhou, Jiangsu 213003, P.R. China
    Copyright: © Cui et al. This is an open access article distributed under the terms of Creative Commons Attribution License.
  • Article Number: 238
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    Published online on: July 6, 2026
       https://doi.org/10.3892/etm.2026.13232
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Abstract

Kidney organoids are important tools for modeling human development and disease, especially in chronic kidney disease (CKD), which is a global health challenge. Current treatment strategies focus on delaying disease progression by managing underlying causes, and in this regard, kidney organoids offer a platform for mechanism‑based therapeutics. Advances in the understanding of human induced pluripotent stem cells (hiPSCs) and sophisticated 3D organ culture methods have enabled researchers to replicate human kidney development and disease mechanisms in vitro, thereby opening new avenues for drug testing. Although the methods for generating renal cell lineages are well established, new protocols for inducing lineages, such as the ureteric bud and collecting ducts, have emerged over the past 5 years. Patient‑derived or genetically edited kidney organoids have been used to successfully model various genetic kidney diseases, notably polycystic kidney disease, and to generate kidney tissues that closely mimic the morphology of real organs. However, achieving more complex disease modeling and generating transplantable synthetic kidneys still has notable challenges. The present review discusses the application of hiPSC‑derived 3D organoids in CKD research and addresses the limitations of current organ culture methods. The present review also examines the impact of CRISPR/Cas9 technology, and investigates potential future directions.
View Figures

Figure 1

Establishment of hiPSC-derived
genetic kidney disease organoid models and their application in
drug screening and validation. The schematic illustrates the
workflow for generating kidney organoid models of hereditary kidney
diseases using patient-derived hiPSCs and CRISPR/Cas9-mediated
genome editing. Somatic cells obtained from patients carrying
disease-associated mutations are reprogrammed into hiPSCs. In
parallel, CRISPR/Cas9 gene editing can be used either to introduce
pathogenic mutations into healthy hiPSCs or to correct
disease-associated mutations through HDR, thereby generating
patient-derived disease hiPSC lines and isogenic control hiPSC
lines. These hiPSCs are subsequently subjected to a kidney
differentiation protocol involving mesoderm induction, nephron
progenitor specification and organoid formation to generate
hiPSC-derived kidney organoids containing nephron-like structures,
including glomeruli, proximal tubules and distal tubules, as well
as podocytes, tubular epithelial cells, endothelial cells, stromal
cells and other renal lineage cells. Disease and control organoids
can then be compared to identify pathological phenotypes, such as
podocyte injury and cyst formation. Diseased organoids may further
be applied in drug repurposing and multiwell plate-based drug
screening, together with phenotype-based readouts, such as
high-content imaging, functional assays, multi-omics analyses and
toxicity assessment, thereby facilitating hit identification, hit
validation, optimization and lead compound selection. hiPSC, human
induced pluripotent stem cell; CRISPR, clustered regularly
interspaced short palindromic repeats; Cas9, CRISPR-associated
protein 9; gRNA, guide RNA; HDR, homology-directed repair.
View References

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Copy and paste a formatted citation
Spandidos Publications style
Cui S, Chen T, Zou Y, Li M, Zhou H, Jiang J and Yang M: Kidney organoids as models for hereditary kidney diseases: Toward precision medicine (Review). Exp Ther Med 32: 238, 2026.
APA
Cui, S., Chen, T., Zou, Y., Li, M., Zhou, H., Jiang, J., & Yang, M. (2026). Kidney organoids as models for hereditary kidney diseases: Toward precision medicine (Review). Experimental and Therapeutic Medicine, 32, 238. https://doi.org/10.3892/etm.2026.13232
MLA
Cui, S., Chen, T., Zou, Y., Li, M., Zhou, H., Jiang, J., Yang, M."Kidney organoids as models for hereditary kidney diseases: Toward precision medicine (Review)". Experimental and Therapeutic Medicine 32.3 (2026): 238.
Chicago
Cui, S., Chen, T., Zou, Y., Li, M., Zhou, H., Jiang, J., Yang, M."Kidney organoids as models for hereditary kidney diseases: Toward precision medicine (Review)". Experimental and Therapeutic Medicine 32, no. 3 (2026): 238. https://doi.org/10.3892/etm.2026.13232
Copy and paste a formatted citation
x
Spandidos Publications style
Cui S, Chen T, Zou Y, Li M, Zhou H, Jiang J and Yang M: Kidney organoids as models for hereditary kidney diseases: Toward precision medicine (Review). Exp Ther Med 32: 238, 2026.
APA
Cui, S., Chen, T., Zou, Y., Li, M., Zhou, H., Jiang, J., & Yang, M. (2026). Kidney organoids as models for hereditary kidney diseases: Toward precision medicine (Review). Experimental and Therapeutic Medicine, 32, 238. https://doi.org/10.3892/etm.2026.13232
MLA
Cui, S., Chen, T., Zou, Y., Li, M., Zhou, H., Jiang, J., Yang, M."Kidney organoids as models for hereditary kidney diseases: Toward precision medicine (Review)". Experimental and Therapeutic Medicine 32.3 (2026): 238.
Chicago
Cui, S., Chen, T., Zou, Y., Li, M., Zhou, H., Jiang, J., Yang, M."Kidney organoids as models for hereditary kidney diseases: Toward precision medicine (Review)". Experimental and Therapeutic Medicine 32, no. 3 (2026): 238. https://doi.org/10.3892/etm.2026.13232
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