43.8:
EPS and iPS Cells in Disease Research
Scientists often use embryonic and induced pluripotent stem cells or EPS and iPS cells in their research.
These stem cells are used in disease modeling and drug screening because they can divide indefinitely without differentiating, and, when stimulated, they can differentiate into most body cell types.
Disease modeling is when cells are used to simulate a specific disease.
In Parkison's disease or PD, neurons of the midbrain are damaged or lost and may not be suitable for disease modeling. Therefore, fibroblasts from a PD patient can be isolated and reprogrammed to form iPS cells.
These cells can differentiate into specific midbrain neurons, which have been used to identify mutations that cause PD and to understand other disease mechanisms.
Scientists also use iPSCs as alternatives to animal models for screening drugs. Patient-derived iPSCs are genetically similar to the patient's own cells.
When these iPSCs differentiate, the cells can be used to test specific drugs for toxicity, efficiency, and the patient's response to the drug.
43.8:
EPS and iPS Cells in Disease Research
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.
Suggested Reading
- Inoue, H., Nagata, N., Kurokawa, H., & Yamanaka, S. (2014). iPS cells: a game changer for future medicine. The EMBO journal, 33(5), 409-417.