ArticleGenes to cells : devoted to molecular & cellular mechanisms2024
Investigation of exon skipping therapy in kidney organoids from Alport syndrome patients derived iPSCs.
Article in Genes to cells : devoted to molecular & cellular mechanisms, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers, 1 of them a synthesis that pooled it.
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Who cites it
9 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Worldwide hotspots and trends in stem cell therapy for kidney disease in the last decade: a bibliometric and visualization analysis from 2015 to 2024.Frontiers in immunology · 2025Pooled it
- Splice modulation of COL4A5 reinstates collagen IV assembly in an organoid model of Alport syndrome.JCI insight · 2026Article
- Advances and future perspectives of kidney organoid technology in renal disease research and clinical translation.Frontiers in medicine · 2026Review
- Advances in organoids for personalized medicine: from technological development to clinical application.Frontiers in cell and developmental biology · 2026Review
- Modeling kidney fibrosis and tubular regeneration in iPSC-derived kidney organoids.Stem cell research & therapy · 2025Article
- Kidney Organoids: Current Advances and Applications.Life (Basel, Switzerland) · 2025Review
- Severe Clinical Phenotype in Alport Syndrome Due to 2Kidney medicine · 2025Article
- A comprehensive splicing characterization of COL4A5 mutations and prognostic significance in a single cohort with X-linked alport syndrome.Frontiers in genetics · 2025Article
- Investigation of exon skipping therapy in kidney organoids from Alport syndrome patients derived iPSCs.Genes to cells : devoted to molecular & cellular mechanisms · 2024Article
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Authors and funding
5 authors.
Funding
Abstract
Alport syndrome (AS) is a hereditary disease caused by mutations in the COL4A5 gene and leads to chronic kidney disease. Currently, no specific treatment has been developed. However, a recent study using AS-model mice demonstrated that the exon skipping method could partially rescue the symptoms. In this study, we evaluated the effects of the exon skipping method using kidney organoids generated from AS-patient-derived induced pluripotent stem cells (AS-iPSCs). We generated kidney organoids from AS-iPSCs, which exhibited nephron structures. As expected, the C-terminus of COL4A5 was not expressed in AS-organoids. Interestingly, anti-sense oligonucleotides restored the expression of the C-terminus of COL4A5 in vitro. Next, we transplanted AS-organoids into mice and evaluated glomerular basement membrane formation in vivo. We found that AS-organoids formed a lower slit diaphragm ratio compared to control organoids. Finally, we assessed the effects of exon skipping on transplanted organoids but observed minimum effects. These studies suggest that AS-iPSCs can generate kidney organoids lacking the C-terminus of COL4A5, and that exon skipping can induce its expression in vitro.
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