Abstract:Background and Aims
Alport Syndrome (AS) is a severe inherited glomerulopathy caused by mutations in the genes encoding the α-chains of type IV collagen, the most abundant component of the glomerular basement membrane (GBM). Alport patients lack effective therapies beyond blockade of the renin-angiotensin system). This work describes the repurposing of two FDA-approved chemical chaperones (4-PBA and TUDCA) to rescue two AS mouse models: a knock-in and a compound heterozygous model bearing the… Show more
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