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Investigation of exon skipping therapy in kidney organoids from Alport syndrome patients derived iPSCs

22 Oct 2024

Abstract excerpt

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...

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PMID: 39435529
DOI: 10.1111/gtc.13170

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Investigation of exon skipping therapy in kidney organoids from Alport syndrome patients derived iPSCsPMID 39435529Read ↗

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Impaired NEPHRIN localization in kidney organoids derived from nephrotic patient iPS cellsSemantic proximity Read ↗

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