Neurodegenerative, neurological, and psychiatric diseases (NNPDs)
significantly contribute to disability and mortality worldwide. Despite numerous
studies, only few effective therapeutic methods and drugs have been found so far even
for the most common diseases, such as Alzheimer’s and Parkinson’s diseases. The lack
of effective therapeutic methods is mainly explainable by the absence of relevant in
vitro model systems to study pathological phenotypes at the cellular and molecular
levels. Induced pluripotent stem cells are a unique source of different types of patientspecific
neurons that can reproduce in vitro the specific features of individual diseases.
In this chapter, the cell models based on patient-specific induced pluripotent stem cellderived
neurons and their use in the search for new drugs and toxicological studies are
described in detail. In addition, the utility of genome engineering for the creation and
study of cell models for NNPDs is discussed.
Keywords: Induced Pluripotent Stem Cells, Neurodegenerative, Neurological and
Psychiatric diseases, Modeling.
