Table 1.

iPSC-based hepatobiliary disease models and their phenotypes.

Type	iPSC-derived disease models	Model phenotypes	References
Hepatocytic	Alpha-1-antitrypsin deficiency	ZAAT polymer accumulation	(Choi et al. 2013; Kaserman and Wilson 2018; Tafaleng et al. 2015)
	Alpers syndrome	Reduced optic atrophy 1 protein	(Li et al. 2015)
	Citrin deficiency	Impaired ureagenesis	(Kim et al. 2016)
	Hemophilia A	FVm deficiency	(Jia et al. 2014; Olgasi et al. 2018)
	Infantile-onset Pompe disease	Lysosomal glycogen accumulation	(Yoshida et al. 2019)
	Liver fibrosis (iPSC-HSC)	Retinyl esters accumulation	(Coll et al. 2018)
	Niemann-pick disease type C	Cholesterol accumulation	(Soga et al. 2015)
	Tangier disease	Impaired cholesterol efflux	(Bi et al. 2017)
	Wilson’s disease	Rapid ATP7B degradation	(Parisi et al. 2018; Yi et al. 2012)
Biliary	Alagille syndrome	Organoid malformation	(Sampaziotis et al. 2015)
	Biliary atresia	Increased fibrosis Reduced biliary differentiation	(Tian et al. 2019)
	Cystic fibrosis	Impaired Cl− channel activity	(Simsek et al. 2016)
	Polycystic liver disease	Cholangiocytic cysts	(Kamiya et al. 2018)