Induced pluripotent stem cells in the study of neurological diseases
1 iPierian, Inc., 951 Gateway Blvd, South San Francisco, CA 94080, USA
2 Department of Neurology, Wayne State University School of Medicine, 421 E Canfield, Room 3209, Detroit, MI 48201, USA
Stem Cell Research & Therapy 2011, 2:37 doi:10.1186/scrt78
This article is part of a review series on Induced pluripotent stem cells. Other articles in the series can be found online at http://stemcellres.com/series/ipscPublished: 21 September 2011
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.