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 |
To model Alagille syndrome.
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 |
To model Citrullinemia type 1.
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 |
To infect organoids from healthy iPSCs with HBV.
To use infected organoids as a model of HBV infection.
|
[160] |