Phenotypic disease-relevant phenotypes developed

Table 10.3 summarizes disease-relevant phenotypes developed from patient-derived iPS cells, which are mainly for neurobiological, liver, and cardiovascular diseases. Another large class that is not included in this review includes diseases notable through their hereditary nature. By the observed phenotypes, all of them show the abilities to recapitulate key aspects of diseases. However, it is still not possible to ensure that the disease models derived from iPSCs demonstrate the similar maturity to those in humans. To date, the disease models show their potential applications in disease-related, toxicity, and differentiation screens. 

Table 10.3 The disease-relevant phenotypes developed by patient-derived iPS cells.

GPCR variant relevance to disease and drug response phenotypes may be useful in modeling systems suitable for drug development— such as targeting such systems as the orexin/hypocretin neuropeptides [37]. Such analyses are, however, complicated by the variability across different populations. 

The penultimate step in drug development is the testing of the drug candidate in animal models of disease, where all the complex interactions that underlie pathophysiological mechanisms take place. It is important that animal models not only manifest the relevant disease phenotype observed in humans, but that the underlying innate and adaptive immune responses are similar to the human disease. In the case of leishmaniasis, there are reasonably good animal models for some, but not all of the cutaneous forms. The animal models for visceral leishmaniasis are less satisfaaory. Nonetheless, the animal models for leishmaniasis are considerably better than those for other parasitic diseases. 

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