Preclinical insights into cholangiopathies: disease modeling and emerging therapeutic targets

Keisaku Sato, Shannon Glaser, Lindsey Kennedy, Suthat Liangpunsakul, Fanyin Meng, Heather Francis, Gianfranco Alpini

Research output: Contribution to journalReview article

3 Scopus citations


Introduction: The common predominant clinical features of cholangiopathies such as primary sclerosing cholangitis (PSC), primary biliary cholangitis (PBC), and biliary atresia (BA) are biliary damage/senescence and liver fibrosis. Curative therapies are lacking, and liver transplantation is the only option. An understanding of the mechanisms and pathogenesis is needed to develop novel therapies. Previous studies have developed various disease-based research models and have identified candidate therapeutic targets. Areas covered: This review summarizes recent studies performed in preclinical models of cholangiopathies and the current understanding of the pathophysiology representing potential targets for novel therapies. A literature search was conducted in PubMed using the combination of the searched term ‘cholangiopathies’ with one or two keywords including ‘model’, ‘cholangiocyte’, ‘animal’, or ‘fibrosis’. Papers published within five years were obtained. Expert opinion: Access to appropriate research models is a key challenge in cholangiopathy research; establishing more appropriate models for PBC is an important goal. Several preclinical studies have demonstrated promising results and have led to novel therapeutic approaches, especially for PSC. Further studies on the pathophysiology of PBC and BA are necessary to identify candidate targets. Innovative therapeutic approaches such as stem cell transplantation have been introduced, and those therapies could be applied to PSC, PBC, and BA.

Original languageEnglish (US)
Pages (from-to)461-472
Number of pages12
JournalExpert Opinion on Therapeutic Targets
Issue number6
StatePublished - Jun 3 2019


  • Cholangiopathy
  • bile duct
  • cholangiocyte
  • inflammation
  • liver fibrosis

ASJC Scopus subject areas

  • Molecular Medicine
  • Pharmacology
  • Drug Discovery
  • Clinical Biochemistry

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