Kibra knockdown inhibits the aberrant Hippo pathway, suppresses renal cyst formation and ameliorates renal fibrosis in nphp1^KO mice.

ABSTRACT:

Introduction

Nephronophthisis (NPH) is an autosomal recessive interstitial cystic kidney disease, which is the most common genetic cause of end-stage renal disease (ESRD) in childhood. The Hippo pathway is regulated by the cilium and has been suggested to be linked to NPH. The aim of the study was to investigate the involvement of Hippo pathway in the pathogenesis of nphp1 defect-associated NPH (NPH1).

Method

Nphp1 knockout (nphp1^KO) Madin-Darby Canine Kidney (MDCK) cells and nphp1^KO C57BL/6J mice were generated via CRISPR gene editing strategy. The siRNAs targeting Kibra, MST1 and LATS1 were designed. An AAV9 vector was designed for Kibra knockdown. The expression and phosphorylation of core Hippo pathway molecules were evaluated. Pathological renal ch

SUBMITTER: Yang Y 

PROVIDER: S-EPMC11850762 | biostudies-literature | 2025 Mar

REPOSITORIES: biostudies-literature