Stem cell contributions to neurological disease modeling and personalized medicine

Prog Neuropsychopharmacol Biol Psychiatry. 2018 Jan 3;80(Pt A):54-62. doi: 10.1016/j.pnpbp.2017.05.025. Epub 2017 May 31.

Abstract

Human induced pluripotent stem cells (iPSCs) represent a revolutionary tool for disease modeling and drug discovery. The generation of tissue-relevant cell types exhibiting a patient's genetic and molecular background offers the ability to develop individual and effective therapies. In this review, we present some major achievements in the neuroscience field using iPSCs and discuss promising perspectives in personalized medicine. In addition to disease modeling, the understanding of the cellular and molecular basis of neurological disorders is explored, including the discovery of new targets and potential drugs. Ultimately, we highlight how iPSC technology, together with genome editing approaches, may bring a deep impact on pre-clinical trials by reducing costs and increasing the success of treatments in a personalized fashion.

Keywords: Disease modeling; Drug screening; Neurological disorders; iPSC.

Publication types

  • Review

MeSH terms

  • Drug Evaluation, Preclinical / methods*
  • Embryonic Stem Cells*
  • Gene Editing / methods*
  • Humans
  • Induced Pluripotent Stem Cells*
  • Models, Neurological*
  • Nervous System Diseases* / therapy
  • Precision Medicine / methods*