Identifying pathological mechanisms underlying human diseases has a key role in discovering novel therapeutic strategies. Animal models have provided valuable tools for modeling human diseases, allowing the identification of pathological mechanisms at distinct developmental stages and in specific cell types in an in vivo setting. Moreover, in mice it is possible to develop in vitro iPSC-based disease models and the corresponding in vivo models in parallel. Comparing the phenotypes observed with corresponding in vitro and in vivo mouse models could provide a better understanding of the strength and limitations of in vitro human iPSC-based models.
