Induced pluripotent stem cells in the study of neurological diseases

Abstract

Five years after their initial derivation from mouse somatic cells, induced pluripotent stem (iPS) cells are an important tool for the study of neurological diseases. By offering an unlimited source of patient-specific disease-relevant neuronal and glial cells, iPS cell-based disease models hold enormous promise for identification of disease mechanisms, discovery of molecular targets and development of phenotypic screens for drug discovery. The present review focuses on the recent advancements in modeling neurological disorders, including the demonstration of disease-specific phenotypes in iPS cell-derived neurons generated from patients with spinal muscular atrophy, familial dysautonomia, Rett syndrome, schizophrenia and Parkinson disease. The ability of this approach to detect treatment effects from known therapeutic compounds has also been demonstrated, providing proof of principle for the use of iPS cell-derived cells in drug discovery.

Figure 1

Human induced pluripotent stem cells can be differentiated into cell types to study neurological disorders. Human induced pluripotent (iPS) stem cells can be differentiated into cell types relevant for the study of neurological disorders. Somatic cells from patients with neurological disorders can be reprogrammed into pluripotent stem cells, which in turn can be differentiated into distinct neuronal and glial cell types, thus offering a human cell platform for mechanistic studies and high-throughput screening for diseases of the central and peripheral nervous system.