Disease-Specific iPS Cell Models in Neuroscience
M. Peitz, J. Jungverdorben and O. Brustle
Affiliation: Institute of Reconstructive Neurobiology, University of Bonn LIFE & BRAIN Center, and LIFE & BRAIN GmbH, Sigmund-Freud Strasse 25, D- 53127 Bonn, Germany.
Keywords: Induced pluripotent stem (iPS) cells, in vitro disease modeling, neurological disorder, neuroscience.
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.
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