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Adeno-associated virus gene therapy prevents progression of kidney disease in genetic models of nephrotic syndrome

9 Aug 2023

Abstract excerpt

Gene therapy for kidney diseases has proven challenging. Adeno-associated virus (AAV) is used as a vector for gene therapy targeting other organs, with particular success demonstrated in monogenic diseases. We aimed to establish gene therapy for the kidney by targeting a monogenic disease of the kidney podocyte. The most common cause of childhood genetic nephrotic syndrome is mutations in the podocyte gene NPHS2...

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PMID: 37556557
DOI: 10.1126/scitranslmed.abc8226

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