Chemical chaperone 4-phenylbutyrate treatment alleviates the kidney phenotype in a mouse model of Alport syndrome with a pathogenic variant in Col4a3.

doi:10.1016/j.kint.2025.05.016

Article

Chemical chaperone 4-phenylbutyrate treatment alleviates the kidney phenotype in a mouse model of Alport syndrome with a pathogenic variant in Col4a3.

Kidney international - 1 Oct 2025

Ioannou Pavlos, Odiatis Christoforos, Hadjisavva Rania, Antoniadou Kyriaki, Pieri Myrtani, Malatras Apostolos, Papagregoriou Gregory, Aristotelous Antrea, Skourides Paris, Samiotaki Martina, Horaček Matija, Ljubanović Danica Galešić, Stylianou Kostas, Deltas Constantinos

Abstract excerpt

INTRODUCTION: Alport Syndrome is a severe inherited glomerulopathy caused by pathogenic variants in genes encoding collagen-IV, the most abundant component of the glomerular basement membrane. Patients with Alport lack effective therapies beyond blockade of the renin-angiotensin-aldosterone system. Here, we test 4-phenylbutyrate (4-PBA) and tauroursodeoxycholic acid (TUDCA), two chemical chaperones, to rescue...

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Chemical chaperone 4-phenylbutyrate treatment alleviates the kidney phenotype in a mouse model of Alport syndrome with a pathogenic variant in Col4a3.

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