Table 2.
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] |