Human kidney organoid generation and disease modeling
Foundational work established human pluripotent stem cell-derived nephron organoids and practical protocols for kidney organoid generation.
Publication highlights
Selected discoveries in human kidney organoids and translational disease modeling.
Selected Discoveries
Research themes that define the platform
Kidney injury and repair in human organoids
Organoid studies identified DNA repair pathways associated with tubular injury response and progression toward chronic kidney disease.
Automated 3D imaging and phenotyping
High-content imaging and AI-assisted analysis support scalable organoid phenotyping for therapeutic discovery.
AAV and gene therapy-associated kidney response
Human kidney organoids can reveal kidney responses to AAV exposure and support gene therapy safety research.
Vascularized organoid and organ-on-chip systems
Flow-based and microphysiological systems extend kidney organoids toward maturation, vascularization, and disease modeling.
Selected papers
Evidence behind the platform
2015
Nephron organoids derived from human pluripotent stem cells model kidney development and injury
Morizane et al. Nature Biotechnology. DOI: 10.1038/nbt.3392. PMID: 26458176.
A foundational paper supporting the core message that human stem cells can be guided into nephron organoids and used to study kidney injury.
2017
Generation of nephron progenitor cells and kidney organoids from human pluripotent stem cells
Morizane and Bonventre. Nature Protocols. DOI: 10.1038/nprot.2016.170. PMID: 28005067.
This protocol paper supports reproducibility and explains how the organoid platform can be implemented as a practical research method.
2019
Organoid vascularization on-chip
Homan et al. Nature Methods. DOI: 10.1038/s41592-019-0325-y.
This paper supports the lab's organoid-on-chip direction by showing how flow-controlled culture can enhance vascularization and maturation in organoid systems.
2022
Modeling injury and repair in kidney organoids reveals that homologous recombination governs tubular intrinsic repair
Gupta, Matsumoto, Hiratsuka et al. Science Translational Medicine. DOI: 10.1126/scitranslmed.abj4772.
This study used kidney organoids to identify DNA repair pathways, including RAD51/FANCD2-mediated repair, as therapeutic targets for preventing progression from acute injury toward chronic kidney disease.
2022
Live functional assays reveal longitudinal maturation of transepithelial transport in kidney organoids
Frontiers in Cell and Developmental Biology. DOI: 10.3389/fcell.2022.978888. PMCID: PMC9420851.
This paper helps position the lab beyond morphology by emphasizing live functional readouts in kidney organoids.
2022
Organoid-on-a-chip model of human ARPKD reveals mechanosensing pathomechanisms for drug discovery
Hiratsuka, Miyoshi, Kroll et al. Science Advances. DOI: 10.1126/sciadv.abq0866.
This ARPKD organoid-on-chip paper supports therapeutic discovery by modeling cystogenesis under flow and identifying disease-relevant mechanosensing pathways and FDA-approved drug repurposing opportunities.
2022
Kidney organoids: a pioneering model for kidney diseases
Tekguc et al. Translational Research. DOI: 10.1016/j.trsl.2022.06.012. PMID: 35750295.
A review framing kidney organoids as platforms for disease modeling, drug screening, organoid-on-chip, and biofabrication.
2023
Immune-infiltrated kidney organoid-on-chip model for assessing T cell bispecific antibodies
Kroll et al. PNAS. DOI: 10.1073/pnas.2305322120. PMID: 37603766.
This is a strong industry-facing example because it connects organoid-on-chip systems with immunotherapy safety and partner-relevant preclinical testing.
2024
Advancing preclinical drug evaluation through automated 3D imaging for high-throughput screening with kidney organoids
Oishi, Tabibzadeh, and Morizane. Biofabrication. DOI: 10.1088/1758-5090/ad38df. PMID: 38547531.
This paper supports an industry message around scalable imaging and high-throughput organoid screening.
2025
AAV for gene therapy drives a nephrotoxic response via NF-kappaB in kidney organoids
Gupta, Zhang, Sabbisetti, Shu, and Morizane. Signal Transduction and Targeted Therapy. DOI: 10.1038/s41392-025-02336-2.
This gene therapy safety paper demonstrates how kidney organoids can reveal human kidney responses to AAV exposure, supporting preclinical assessment for renal gene therapy strategies.
2026
Deciphering the impact of RAC1-SPTAN1 in ARPKD cystogenesis using multifaceted models
Kuraoka et al. Advanced Science. DOI: 10.1002/advs.202524001.
This ARPKD study extends therapeutic discovery using multifaceted disease models and supports a patent-pending approach to ARPKD therapy development.
References
- PubMed: Morizane et al., Nature Biotechnology, 2015
- PubMed: Morizane and Bonventre, Nature Protocols, 2017
- PMC: Gupta et al., Science Translational Medicine, 2022
- PubMed: Hiratsuka et al., Science Advances, 2022
- PubMed: Tekguc et al., Translational Research, 2022
- PMC: Kroll et al., PNAS, 2023
- PubMed: Oishi et al., Biofabrication, 2024
- Nature: Gupta et al., Signal Transduction and Targeted Therapy, 2025
- Mayo Clinic Pure: Kuraoka et al., Advanced Science, 2026