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Exploration of Gene Therapy for Alport Syndrome

23 May 2024

Abstract excerpt

Alport syndrome is a hereditary disease caused by mutations in the genes encoding the alpha 3, alpha 4, and alpha 5 chains of type IV collagen. It is characterized by hematuria, proteinuria, progressive renal dysfunction, hearing loss, and ocular abnormalities. The main network of type IV collagen in the glomerular basement membrane is composed of α3α4α5 heterotrimer. Mutations in these genes can lead to the...

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PMID: 38927366
DOI: 10.3390/biomedicines12061159

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