What are Organoids?

Organoids are miniaturized organs that are derived from stem cells in vitro. They are formed as three-dimensional tissue-like structures by utilizing the self-renewal and multi-differentiation capacities of stem cells.

Compared to conventional cultured cells and spheroids, organoids are anatomically and functionally close to the organs in the living body. Organoids can be constructed from both normal and diseased tissues, allowing for utilization in various research applications. These tissue structures are used for basic research such as embryology, physiology, and evolution as well as more involved research including characterizing pathological disease conditions and drug discovery research. In addition, there are interesting studies using applied research for regenerative medicine using organoids for transplantation.

Types and Applications of Organoids

Organoids are mainly derived from pluripotent stem cells (iPS cells, ES cells) and organoids derived from tissue stem cells.

Utilization of organoid technology

Organoid technology is utilized in research areas such as embryology and the investigation of disease mechanisms. Organoids also used in drug discovery research for applications such as drug screening and regenerative medicine. The use of organoids has allowed for important downstream discoveries for better physiological understanding in order to improve human health and body.

Schematic diagram of organoid technology

Applications of pluripotent stem cell organoids

Research on organoids derived from pluripotent stem cells has elucidated the differentiation mechanism of each organ, leading to the investigation of disease mechanisms, drug screening, and application to regenerative medicine.

Table 4
human iPSC-derived organoids for modeling development and disease

Starting cells	Organoids	Applications	References
Human ESCs and iPSCs	Cerebral organoids	Modeling human cortical development and microcephaly	Lancaster et al. 2013¹¹⁷
Human ESCs and iPSCs	Cerebral organoids	Modeling human fetal neocortex development	Camp et al. 2015¹⁰⁷
Human iPSCs	Brain organoids	Modeling autism spectrum disorders	Mariani et al. 2015¹⁰⁸
Human ESCs and iPSCs	Brain organoids	Modeling Zika virus infection	Cugola et al. 2016¹⁰⁹
Human iPSCs	Brain-region-specific organoids	Modeling Zika virus infection. Future applications include modeling human brain development and diseases and drug testing.	Qian et al. 2016¹¹⁰
Human iPSCs	Brain organoids	Modeling Zika virus infection	Garcez et al. 2016¹¹¹
Human iPSCs	Brain organoids	Modeling Seckel syndrome	Gabriel et al. 2016¹¹²
Human ESCs and iPSCs	Cortical organoids	Modeling species difference	Otani et al. 2016¹¹³
Human iPSCs	Cortical spheroids	Modeling cortical development and diseases	Pasca et al. 2015¹¹⁴
Human iPSCs	Retinal organoids	Modeling glaucoma	Tucker et al. 2014¹⁰⁶
Human ESCs and iPSCs	Intestinal organoids	Studying intestinal development. Future applications include intestinal disease modeling.	Spence et al. 2011¹⁰³
Human ESCs and iPSCs	Small intestinal organoids	Transplantation to demonstrate responsiveness to systemic signals. Future applications include studying intestinal development and diseases.	Watson et al. 2014¹⁰⁵
Human iPSC	Liver bud	Organ-bud transplantation for regenerative medicine	Takebet et al. 2013⁹⁹
Human iPSCs	Cystic organoids	Modeling Alagille syndrome, polycystic liver disease and cystic fibrosis-associated cholangiopathy and drug validation	Sampaziotis et al. 2015¹⁰⁰
Human iPSCs	Cystic organoids	Studying biliary development and cystic fibrosis	Ogawa et al. 2015¹⁰¹
Human iPSC	Lung organoids	Studying lung development. Future applications include lung disease modeling.	Dye et al. 2015¹⁰²
Human iPSCs	Kidney organoids	Future applications include kidney disease modeling, nephrotoxicity screening and cell therapy.	Takasato et al. 2015¹⁰⁴
Human ESCs and iPSCs	Gastric organoids	Modeling gastric development and H. pylori infection	McCracken et al. 2016⁹⁸

Adapted from Table 4 of Yanhong Shi et al. “Induced pluripotent stem cell technology: a decade of progress”
Nat Rev Drug Discov. Vol. 16 no.2 (2017) : 115–130.