ArticleJournal of translational medicine2023
Modeling of Fabry disease nephropathy using patient derived human induced pluripotent stem cells and kidney organoid system.
Article in Journal of translational medicine, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers.
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Who cites it
19 citing papers in PubMed, 22 citations in OpenAlex.
- Kidney organoids as models for hereditary kidney diseases: Toward precision medicine (Review).Experimental and therapeutic medicine · 2026Review
- Modeling Fabry nephropathy: insights from experimental systems towards precision nephrology.Disease models & mechanisms · 2026Review
- Organoids for Metabolic Disease Modeling.Journal of inherited metabolic disease · 2026Review
- Patients' perception of proton pump inhibitors use and their risks.World journal of clinical oncology · 2026Article
- Organoids in Cancer Research and Regenerative Medicine: Current Status, Challenges, and Future Prospects.MedComm · 2026Review
- Kidney Organoids: Current Advances and Applications.Life (Basel, Switzerland) · 2025Review
- Modeling of kidney allograft rejection using hiPSC-derived kidney organoids and HLA-mismatched PBMCs: an in vitro co-culture system.Cellular and molecular life sciences : CMLS · 2025Article
- CRISPR and gene editing for kidney diseases: where are we?Clinical kidney journal · 2025Review
- Current status and future prospects of toxicity assessment using organoids.Toxicological research · 2025Review
- Article
- Human in vitro models for Fabry disease: new paths for unravelling disease mechanisms and therapies.Journal of translational medicine · 2024Review
- Kidney organoids: steps towards better organization and function.Biochemical Society transactions · 2024Review
- Clinical characteristics and induced pluripotent stem cells (iPSCs) disease model of Fabry disease caused by a novel GLA mutation.QJM : monthly journal of the Association of Physicians · 2024Article
- Kidney Organoid Modeling of WT1 Mutations Reveals Key Regulatory Paths Underlying Podocyte Development.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2024Article
- Review
- Fetal Kidney Grafts and Organoids from Microminiature Pigs: Establishing a Protocol for Production and Long-Term Cryopreservation.International journal of molecular sciences · 2024Article
- Trends and challenges in organoid modeling and expansion with pluripotent stem cells and somatic tissue.PeerJ · 2024Review
- Modeling ofCells · 2023Article
- Progress of Induced Pluripotent Stem Cell-Derived Renal Organoids in Clinical Application.Kidney diseases (Basel, Switzerland)Review
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Authors and funding
12 authors at 5 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectivesTo explore the possibility of kidney organoids generated using patient derived human induced pluripotent stem cells (hiPSC) for modeling of Fabry disease nephropathy (FDN).
methodsFirst, we generated hiPSC line using peripheral blood mononuclear cells (PBMCs) from two male FD-patients with different types of GLA mutation: a classic type mutation (CMC-Fb-001) and a non-classic type (CMC-Fb-003) mutation. Second, we generated kidney organoids using wild-type (WT) hiPSC (WTC-11) and mutant hiPSCs (CMC-Fb-001 and CMC-Fb-003). We then compared alpha-galactosidase A (α-GalA) activity, deposition of globotriaosylceremide (Gb-3), and zebra body formation under electromicroscopy (EM).
resultsBoth FD patients derived hiPSCs had the same mutations as those detected in PBMCs of patients, showing typical pluripotency markers, normal karyotyping, and successful tri-lineage differentiation. Kidney organoids generated using WT-hiPSC and both FD patients derived hiPSCs expressed typical nephron markers without structural deformity. Activity of α-GalA was decreased and deposition of Gb-3 was increased in FD patients derived hiPSCs and kidney organoids in comparison with WT, with such changes being far more significant in CMC-Fb-001 than in CMC-Fb-003. In EM finding, multi-lammelated inclusion body was detected in both CMC-Fb-001 and CMC-Fb-003 kidney organoids, but not in WT.
conclusionsKidney organoids generated using hiPSCs from male FD patients might recapitulate the disease phenotype and represent the severity of FD according to the GLA mutation type.
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