Embryonic and induced pluripotent stem cells are excellent models for disease research because of their ability to self-renew and differentiate into most cell types. Somatic cells from a patient are isolated and reprogrammed into induced pluripotent stem cells or iPSCs. These iPSCs are later differentiated into the desired cell type, which mirrors the diseased cell of the patient. In this way, disease models have been created for investigating diseases such as Down syndrome, type I diabetes, and spinal muscular atrophy.

Drugs are usually tested in animal models, but using animals for research is expensive and may raise ethical concerns. Additionally, animal models may not accurately mirror human physiology or the disease under investigation. Patient-derived iPSCs have the same genetic makeup as the patient’s disease-affected cells. When differentiated, these cells can replicate the physiology of the diseased tissue and show a nearly accurate disease phenotype.

Drugs for treating cardiovascular, neurodegenerative, and liver disorders are currently being tested using cells derived from iPSCs. The cells are used to screen drugs for their effects and possible toxicity at varying doses. This can be part of a pre-clinical study of a medicine before it is tested directly in humans in clinical trials.