Disease-Specific iPS Cell Models in Neuroscience
M. Peitz, J. Jungverdorben and O. Brustle
Pages 832-841 (10)
Neurodegenerative diseases are a heterogeneous group of sporadic or familial disorders of the
nervous system that mostly lead to a progressive loss of neural cells. A major challenge in studying the
molecular pathomechanisms underlying these disorders is the limited experimental access to disease-affected
human nervous system tissue. In addition, considering that the molecular disease initiation occurs years or
decades before the symptomatic onset of a medical condition, these tissues mostly reflect only the final phase
of the disease. To overcome these limitations, various model systems have been established based on gainand
loss-of-function studies in transformed cell lines or transgenic animal models. Although these approaches
provide valuable insights into disease mechanisms and development they often lack physiological protein
expression levels and a humanized context of molecular interaction partners. The generation of human
induced pluripotent stem (hiPS) cells from somatic cells provides access to virtually unlimited numbers of
patient-specific cells for modeling neurological disorders in vitro. In this review, we focus on the current
progress made in hiPS cell-based modeling of neurodegenerative diseases and discuss recent advances in
the quality assessment of hiPS cell lines.
Induced pluripotent stem (iPS) cells, in vitro disease modeling, neurological disorder, neuroscience.
Institute of Reconstructive Neurobiology, University of Bonn LIFE & BRAIN Center, and LIFE & BRAIN GmbH, Sigmund-Freud Strasse 25, D- 53127 Bonn, Germany.