Kidney organoids in translational research: disease modeling, drug discovery, and unresolved challenges

Nov 5, 2025Cell and tissue research

Kidney organoids for studying diseases, testing drugs, and remaining challenges

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Regenerative Health on OpenScience ↗PubMed ↗DOI ↗

Abstract

Kidney organoids derived from human pluripotent stem cells effectively model monogenic renal disorders and drug-induced nephrotoxicity.

These organoids replicate key human kidney structures, including nephron-like features and glomeruli.
They exhibit greater cellular diversity compared to traditional two-dimensional cell cultures.
Kidney organoids can model various renal disorders such as autosomal dominant polycystic kidney disease and congenital nephrotic syndrome.
They respond in a dose- and time-dependent manner to nephrotoxic drugs like cisplatin and tenofovir.
Challenges remain regarding their vascularization, developmental maturity, and variability in reproducibility.
Innovative bioengineering approaches aim to improve the functionality and reliability of kidney organoids.

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Kidney organoids derived from human pluripotent stem cells (hPSCs) have emerged as powerful platforms for translational nephrology, enabling complex renal pathophysiology modeling in physiologically relevant three-dimensional contexts. This review synthesizes recent advances in kidney organoid applications for disease modeling and drug discovery, highlighting their translational potential beyond developmental biology. These organoids recapitulate key human kidney architectural features, including nephron-like structures with glomeruli and tubules, while exhibiting greater cellular heterogeneity than traditional two-dimensional cultures. They effectively model monogenic renal disorders including autosomal dominant polycystic kidney disease (ADPKD), congenital nephrotic syndrome, and Alport syndrome, as well as acquired conditions like acute kidney injury and drug-induced nephrotoxicity. Kidney organoids serve as predictive nephrotoxicity screening platforms, demonstrating dose- and time-dependent responses to cisplatin, tenofovir, and aristolochic acid. However, significant challenges persist, including insufficient vascularization, developmental immaturity, segmental bias, absent urinary drainage systems, and reproducibility variability. Emerging bioengineering strategies-including endothelial co-culture, microfluidic integration, and 3D bioprinting-aim to address these limitations. Integrating stem cell biology with engineering innovations and multi-omics technologies will be crucial for refining kidney organoids into scalable, reproducible models that faithfully recapitulate human kidney physiology and disease, ultimately enabling their translation into precision medicine applications.

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Kidney organoids in translational research: disease modeling, drug discovery, and unresolved challenges

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