TABLE 2.
Unique advantages of organoids in hereditary disease.
| Report year | Cellular input | Organoids | Disease | Applications |
|---|---|---|---|---|
| 2013 | Murine and human intestinal stem cells | Epithelial organoids | Cystic fibrosis | Disease model; CRISPR-based genome editing; Gene therapy Schwank et al. (2013) |
| 2015 | Human rectal stem cell | Intestinal organoids | Cystic fibrosis | Disease model; Gene therapy Vidović et al. (2016) |
| 2016 | Human iPSC | Optic cup organoids | Leber congenital amaurosis | Disease model; Pathological mechanism; Drug screening Parfitt et al. (2016) |
| 2017 | Mouse Liver tissues | Liver organoids | Alagille syndrome | Verify the characteristics of the Jag1Ndr/Ndr mouse model Andersson et al. (2018) |
| 2017 | Human iPSC | Forebrain-type organoids | Miller-Dieker syndrome | Disease model; Pathological mechanism; Modeling of cell-cell communication Iefremova et al. (2017) |
| 2017 | Human iPSC | Cerebral Organoids | Miller-Dieker syndrome | Disease model; Pathological mechanism; Single-cell RNA sequencing Bershteyn et al. (2017) |
| 2017 | Human iPSC | Hepatic organoids | Alagille syndrome | Disease model; CRISPR-based genome editing; Gene therapy Guan et al. (2017) |
| 2018 | Human iPSC | Cerebral organoids | Sandhoff disease | Disease model; Pathological mechanism Allende et al. (2018) |
| 2018 | Human iPSC | Retinal organoids | Retinitis pigmentosa | Disease model; CRISPR-based genome editing; gene therapy Deng et al. (2018) |
| 2018 | Human iPSCs | Retinal organoids | Leber congenital amaurosis type 10 | Drug screening Dulla et al. (2018) |
| 2019 | Human iPSCs | Cerebral organoids | Juvenile form of neuronal ceroid lipofuscinosis | Disease model and pathological mechanism Gomez-Giro et al. (2019) |
| 2019 | Human iPSCs | Proximal lung organoids | Cystic fibrosis | Disease model, CRISPR-based genome editing, and gene therapy Ruan et al. (2019) |
| 2019 | Human primary rectal stem cells | Intestinal organoids | Cystic fibrosis | Disease model, CRISPR-based genome editing, and gene therapy Maule et al. (2019) |
| 2020 | Human iPSCs | Cerebral organoids | Schizophrenia | Disease model and pathological mechanism Benson et al. (2020) |
| 2020 | Human iPSCs | Retinal organoids | X-linked retinitis pigmentosa | Disease model, pathological mechanism, and CRISPR-based genome editing Lane et al. (2020) |
| 2020 | Human intestinal stem cells | Intestinal organoids and airway organoids | Cystic fibrosis | Organoid biobank, adenine-based editing gene therapy Geurts et al. (2020) |
iPSCs, induced pluripotent stem cell.