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. 2021 Sep 28;22(19):10471. doi: 10.3390/ijms221910471

Table 2.

Summary of hPSC-derived liver disease models.

Diseases Modeling Strategies Applications
Familial hypercholesterolemia HLCs from patient-derived iPSCs High-throughput drug screening [47];
Preclinical drug efficacy evaluation [48]
mtDNA depletion syndrome HLCs and liver organoids from patient-derived iPSCs and gene-corrected counterparts Disease mechanism investigation [49]
HLCs from genetically engineered iPSCs
(DGUOK deficient)
High-throughput drug screening [50]
Wilson’s disease HLCs from genetically engineered hPSCs
(mutations in the ATP7B gene)
Preclinical drug efficacy evaluation [51];
Disease features modeling [52]
Primary hyperoxaluria type 1 HLCs from genetically
corrected patient-derived iPSCs
Therapeutical gene correction [53]
Congenital hepatic fibrosis HLCs from genetically engineered iPSCs
(PKHD1 knockout)
Disease mechanism investigation [54]
Urea cycle disorders Liver organoids from patient-derived iPSCs andthe gene-corrected counterpart Therapeutical gene correction [55]
Liver steatosis HLCs cultured in monolayers Disease mechanism investigation [56]
Liver organoids Anti-steatosis drug screening [57];
Disease features modeling [34]
Multicellular liver organoids
from patient-derived iPSCs
Modeling the progressive features of steatohepatitis [43]
Liver organoids-on-a-chip system Modeling the features of fatty liver using bioengineered systems [58]
Biofabricated human fatty liver tissue
with SIRT1 knock down hiPSC-derived HLCs
Investigating the effects of a specific gene [59]
HBV infection HLCs infected with HBV Novel antiviral agent identification [60]
Liver organoids infected with HBV Recapitulating virus life cycle and hepatic dysfunction in 3D organoids [61]
Chimeric mice engrafted with hiPSC-HLCs Antivirals evaluation in vitro and in vivo [62]
HCV infection Chimeric mice engrafted with
hPSC-derived hepatic lineages
Long-term infection of multiple HCV genotypes [63]