Article

Increased <i>in vivo</i> transduction of AAV-9 cargo in Alport podocytes

2025-04-28

Abstract excerpt

Alport syndrome is a rare genetic disorder characterized by progressive kidney disease, hearing loss, and eye abnormalities. Gene therapy for Alport syndrome has not yet been realized due to technical challenges, including effective transduction of target cells. In this study, we established a quantitative testing platform using podocytes in culture, ex vivo glomeruli, and a mouse model of Alport syndrome to eval...

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Literature Corpus work: 1908118e-bf49-5a66-88b8-44bf21d1054d
DOI: 10.1101/2025.04.28.650965

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