Article

Increased in vivo transduction of AAV-9 cargo in Alport podocytes

2025-07-30

Abstract excerpt

Background Alport syndrome is a rare genetic disorder characterized by progressive kidney disease, hearing loss, and eye abnormalities. It is caused by variants in the COL4A3 , COL4A4 or COL4A5 genes, which disrupt the synthesis, secretion and assembly of the alpha-3, -4 and -5 chains of type IV collagen. The defective collagen IV network results in the formation of an abnormal glomerular basement membrane (GBM...

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Literature Corpus work: 26ff0946-bc1a-5cee-8a88-180636a1ec3f
DOI: 10.12688/wellcomeopenres.24212.1

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