• Human biology and disease modelling: Organoids have demonstrated significant utility in modelling and investigating common and rare diseases in various organs.
– For example, cystic fibrosis is a genetic disorder caused by mutations in the gene CFTR causing multi-organ dysfunctions affecting the lungs, pancreas, liver and intestine. Cystic fibrosis organoid models from mouse models or patients have been developed from the colon, liver, rectum, the respiratory system, and the small intestine. These organoids were used for investigating disease biology, mirroring patients’ drug responses, and gene therapy to rescue CFTR mutations using CRISPR–Cas9.
– Organoids have been also used to study infectious diseases. For example, studying the effects of the SARS-CoV-2 virus on lung organoids for mechanistic and drug screenings purposes.
• Tissue engineering and regenerative medicine: Tissue-derived organoids can be a potential source of transplantable material for regenerative medicine. For example, pancreatic islets cultured in an artificial ECM could be successfully engrafted into diabetic mouse models, rescuing insulin production and reversing hyperglycaemia.
• Drug screening and discovery: For example, screening of 240 tyrosine kinase inhibitors on organoids derived from patients with ovarian or peritoneal tumours provided individualized drug sensitivity profiles within a week from surgery.
• Personalized medicine: tailoring therapies to each patient using patient-specific organoids.