# Technological Innovations and the Translational Path of Kidney Organoids

**Authors:** Anni Li, Zhonglin Chai, Karin Jandeleit-Dahm, Jay C. Jha

PMC · DOI: 10.3390/biomedicines14020327 · Biomedicines · 2026-01-31

## TL;DR

This paper reviews how kidney organoids, derived from stem cells, can model kidney development and disease, and their potential for drug testing and regenerative medicine.

## Contribution

The paper systematically reviews key advancements and translational challenges in kidney organoid technology.

## Key findings

- Kidney organoids replicate kidney cellular interactions and functions, enabling disease modeling and drug screening.
- Optimized stem cell differentiation and bioengineering have improved organoid development and applications.
- Translational challenges include vascularization, functional maturity, and scalable production.

## Abstract

Kidney organoids, as three-dimensional microstructures derived from human pluripotent stem cells or adult stem cells, precisely simulate the cellular heterogeneity, spatial conformation, and some physiological functions of human kidney units in vitro. Kidney organoids are three-dimensional microstructures derived from human pluripotent stem cells (hPSCs). They precisely simulate the cellular heterogeneity, spatial conformation, and key physiological functions of human kidney units in vitro. This technology, by replicating the interaction network between the glomerulus and renal tubules, provides an unprecedented window for observing the dynamic development and pathological processes of human kidneys. This technology replicates the interaction network between the glomerulus and renal tubules. It thereby provides an unprecedented window into human kidney development and disease. Based on the strong similarity between organoids and native organs, as well as the human genetic information they carry, both iPSC-derived and patient-specific organoids have demonstrated significant value in kidney disease modeling, drug toxicity testing, and the development of regenerative treatment strategies. This review systematically elucidates the key advancements in the field of kidney organoids, including optimized strategies for stem cell-directed differentiation, innovations in culture systems driven by biomaterials engineering, technological breakthroughs in disease model construction, and applications of organoids in drug screening platforms and regenerative medicine. Additionally, it analyzes translational challenges such as the lack of vascularization, insufficient functional maturity, and obstacles in standardized production. These insights will deepen the understanding of kidney pathological mechanisms and propel organoid technology towards substantial clinical therapeutic applications. This review summarizes how convergent technologies in stem cell biology and bioengineering aim to bridge this functional gap. We examine the use of advanced organoids in disease modeling and drug discovery. We also highlight their current limitations. Our focus is on the core translational bottlenecks: vascularization, long-term maturation, and scalable production. Overcoming these hurdles is essential to transform kidney organoids from a research tool into a platform for precision medicine and regenerative therapy.

## Full-text entities

- **Genes:** EPO (erythropoietin) [NCBI Gene 2056] {aka DBAL, ECYT5, EP, MVCD2}, EPAS1 (endothelial PAS domain protein 1) [NCBI Gene 2034] {aka ECYT4, HIF2A, HLF, MOP2, PASD2, bHLHe73}, Egfr (epidermal growth factor receptor) [NCBI Gene 13649] {aka 9030024J15Rik, Erbb, Errb1, Errp, Wa5, wa-2}, BMP7 (bone morphogenetic protein 7) [NCBI Gene 655] {aka OP-1}, Agt (angiotensinogen) [NCBI Gene 11606] {aka AngI, AngII, Aogen, Serpina8}, SIX1 (SIX homeobox 1) [NCBI Gene 100156847], SMAD3 (SMAD family member 3) [NCBI Gene 4088] {aka HSPC193, HsT17436, JV15-2, LDS1C, LDS3, MADH3}, PTH (parathyroid hormone) [NCBI Gene 5741] {aka FIH1, PTH1}, Golm1 (golgi membrane protein 1) [NCBI Gene 105348] {aka 2310001L02Rik, D030064E01Rik, GP73, Golph2, PSEC0257}, SIX2 (SIX homeobox 2) [NCBI Gene 100525814], ACTA1 (actin alpha 1, skeletal muscle) [NCBI Gene 58] {aka ACTA, ASMA, CFTD, CFTD1, CFTDM, CMYO2A}, Fgf9 (fibroblast growth factor 9) [NCBI Gene 14180] {aka Eks, FGF-9, Fgf4b, GAF, HBGF-9}, Ace2 (angiotensin converting enzyme 2) [NCBI Gene 70008] {aka 2010305L05Rik}, Fn1 (fibronectin 1) [NCBI Gene 14268] {aka E330027I09, Fn, Fn-1}, COL4A5 (collagen type IV alpha 5 chain) [NCBI Gene 1287] {aka ASLN, ATS, ATS1, CA54}, Col1a1 (collagen, type I, alpha 1) [NCBI Gene 12842] {aka Col1a-1, Cola-1, Cola1, Mov-13, Mov13}, NPHS1 (NPHS1 adhesion molecule, nephrin) [NCBI Gene 4868] {aka CNF, NPHN, nephrin}, Yap1 (yes-associated protein 1) [NCBI Gene 22601] {aka Yap, Yap65, Yki, Yorkie}, LCN2 (lipocalin 2) [NCBI Gene 3934] {aka 24p3, MSFI, NGAL, p25}, BAK1 (BCL2 antagonist/killer 1) [NCBI Gene 578] {aka BAK, BAK-LIKE, BCL2L7, CDN1}, TGFB1 (transforming growth factor beta 1) [NCBI Gene 7040] {aka CAEND1, CED, DPD1, IBDIMDE, LAP, TGF-beta1}, TAFAZZIN (tafazzin, phospholipid-lysophospholipid transacylase) [NCBI Gene 6901] {aka BTHS, CMD3A, EFE, EFE2, G4.5, LVNCX}, Alb (albumin) [NCBI Gene 11657] {aka Alb-1, Alb1, BCL001, BCL002, BPL001}, PKD1 (polycystin 1, transient receptor potential channel interacting) [NCBI Gene 5310] {aka PBP, PC1, Pc-1, TRPP1, eliosin}, Havcr1 (hepatitis A virus cellular receptor 1) [NCBI Gene 171283] {aka KIM-1, TIM-1, Tim1, Timd1}, Synpo (synaptopodin) [NCBI Gene 104027] {aka 9030217H17Rik, 9130229N11, 9330140I15Rik}, Acta2 (actin alpha 2, smooth muscle, aorta) [NCBI Gene 11475] {aka 0610041G09Rik, Actvs, SMAalpha, SMalphaA, a-SMA, alphaSMA}, Mapk14 (mitogen-activated protein kinase 14) [NCBI Gene 26416] {aka CSBP2, Crk1, Csbp1, Mxi2, PRKM14, PRKM15}, NR1H4 (nuclear receptor subfamily 1 group H member 4) [NCBI Gene 9971] {aka BAR, FXR, HRR-1, HRR1, PFIC5, RIP14}, Tgfb1 (transforming growth factor, beta 1) [NCBI Gene 21803] {aka TGF-beta1, TGFbeta1, Tgfb, Tgfb-1}, BAX (BCL2 associated X, apoptosis regulator) [NCBI Gene 581] {aka BCL2L4}, CD70 (CD70 molecule) [NCBI Gene 970] {aka CD27-L, CD27L, CD27LG, LPFS3, TNFSF7, TNLG8A}, Pparg (peroxisome proliferator activated receptor gamma) [NCBI Gene 19016] {aka Nr1c3, PPAR-gamma, PPAR-gamma2, PPARgamma, PPARgamma2}, VHL (von Hippel-Lindau tumor suppressor) [NCBI Gene 7428] {aka HRCA1, RCA1, VHL1, pVHL}, SALL1 (spalt like transcription factor 1) [NCBI Gene 100519899]
- **Diseases:** acute (MESH:D000208), PKD (MESH:D007690), ADPKD (MESH:D016891), kidney-deficient (MESH:D007680), developmental disorders (MESH:D002658), cystic diseases (MESH:C563237), ischemia (MESH:D007511), DKD (MESH:D003928), metabolic diseases (MESH:D008659), genetic defects (MESH:D030342), hypoxia (MESH:D000860), Cyst (MESH:D003560), CD (MESH:D002292), Kidney Diseases (MESH:D007674), fibrotic drugs (MESH:D000081015), collagen metabolism disorders (MESH:D003095), AKI (MESH:D058186), diabetes (MESH:D003920), ESRD (MESH:D007676), tumor (MESH:D009369), tubulointerstitial and glomerular diseases (MESH:C536137), CKD (MESH:D051436), cytotoxicity (MESH:D064420), Fibrosis (MESH:D005355), autosomal recessive polycystic kidney disease (MESH:D017044), injury to (MESH:D014947), inflammation (MESH:D007249), Alport syndrome (MESH:D009394), viral infection (MESH:D014777), hypertensive (MESH:D006973), acute injury (MESH:D001930)
- **Chemicals:** glycosides (MESH:D006027), glucose (MESH:D005947), creatinine (MESH:D003404), Minoxidil (MESH:D008914), Imatinib (MESH:D000068877), CHIR99021 (MESH:C473711), urea (MESH:D014508), aldosterone (MESH:D000450), cAMP (-), cisplatin (MESH:D002945), oxygen (MESH:D010100)
- **Species:** Sus scrofa (pig, species) [taxon 9823], Gallus gallus (bantam, species) [taxon 9031], Mus musculus (house mouse, species) [taxon 10090], Rattus norvegicus (brown rat, species) [taxon 10116], Homo sapiens (human, species) [taxon 9606]
- **Mutations:** R2430X

## Full text

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## References

60 references — full list in the complete paper: https://tomesphere.com/paper/PMC12937646/full.md

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Source: https://tomesphere.com/paper/PMC12937646