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. 2020 Aug 27;21(17):6215. doi: 10.3390/ijms21176215

Table 3.

Summary of studies on liver organoid applications as disease modeling.

Author Approach Disease model Gene Aim Reference
Sampaziotis et al. Human iPSCs from healthy donors and cystic fibrosis patients were differentiated into cholangiocyte-like cells Cystic fibrosis associated biliary disease Cystic fibrosis transmembrane conductance regulator gene (CFTR) To test the effects of the drug VX809 on organoids [51,157]
Guan et al. iPSCs from healthy donors and Alagille syndrome patients were differentiated into 3D human hepatic organoids Alagille syndrome JAG1
  1. To model Alagille syndrome.

  2. To introduce and revert JAG1 mutation with CRISPR/Cas9 technology and piggyBac transposon system.

[126]
Akbani et al. iPSC-derived-EpCAM-positive endodermal cells differentiated into hepatic organoids Citrullinemia type 1 Argininosuccinate synthetase (ASS1) gene
  1. To model Citrullinemia type 1.

  2. To introduce the functional form of ASS1 by lentiviral vector transduction

[158]
Nie et al. Human iPSC-derived endodermal, mesenchymal, and endothelial cells were cultured in specific medium to obtain liver organoids Hepatitis B virus (HBV) infection ND
  1. To infect organoids from healthy iPSCs with HBV.

  2. To use infected organoids as a model of HBV infection.

[160]