PUBLICATION

Zebrafish ppp1r21 mutant as a model for the study of primary biliary cholangitis

Authors
Wu, C., Zhang, W., Luo, Y., Cheng, C., Wang, X., Jiang, Y., Li, S., Luo, L., Yang, Y.
ID
ZDB-PUB-230605-33
Date
2023
Source
Journal of genetics and genomics = Yi chuan xue bao   50(12): 1004-1013 (Journal)
Registered Authors
Luo, Lingfei, Yang, Yun
Keywords
PDC-E2, PI3K/AKT/mTOR pathway, Zebrafish mutant, ppp1r21, primary biliary cholangitis
MeSH Terms
  • Animals
  • Dogs
  • Liver Cirrhosis, Biliary*/metabolism
  • Liver Cirrhosis, Biliary*/pathology
  • Phosphatidylinositol 3-Kinases/genetics
  • Proto-Oncogene Proteins c-akt/genetics
  • TOR Serine-Threonine Kinases
  • Zebrafish
PubMed
37271428 Full text @ J. Genet. Genomics
Abstract
Primary biliary cholangitis (PBC) is an autoimmune cholestatic liver disease that progresses to fibrosis and cirrhosis, resulting from the gradual destruction of intrahepatic bile ducts. Exploring genetic variants associated with PBC is essential to understand the pathogenesis of PBC. Here we identify a zebrafish balloon dog (blg) mutant with intrahepatic bile duct branching defects, exhibiting several key pathological PBC-like features, including immunodominant autoantigen PDC-E2 production, cholangiocyte apoptosis, immune cell infiltration, inflammatory activation, and liver fibrosis. blg encodes the protein phosphatase 1 regulatory subunit 21 (Ppp1r21), which is enriched in the liver and its peripheral tissues and plays a vital role in the early intrahepatic bile duct formation stage. Further studies show an excessive activation of the PI3K/AKT/mTOR pathway in the hepatic tissues in the mutant, while treatment with the pathway inhibitor LY294002 and rapamycin partially rescues intrahepatic bile duct branching defects and alleviate the PBC-like symptoms. These findings implicate the potential role of the Ppp1r21-mediated PI3K/AKT/mTOR pathway in the pathophysiology of PBC.
Genes / Markers
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Phenotype
Mutations / Transgenics
Human Disease / Model
Sequence Targeting Reagents
Fish
Antibodies
Orthology
Engineered Foreign Genes
Mapping