4.6 Review

Stem cell contributions to neurological disease modeling and personalized medicine

Publisher

PERGAMON-ELSEVIER SCIENCE LTD
DOI: 10.1016/j.pnpbp.2017.05.025

Keywords

iPSC; Disease modeling; Drug screening; Neurological disorders

Funding

  1. Fundacao de Amparo a Pesquisa do Estado de Sao Paulo (Sao Paulo Research Foundation FAPESP) [2012/50880-4]
  2. Conselho Nacional de Desenvolvimento Cientifico e Tecnologico (CNPq), Brazil
  3. NARSAD
  4. NIH [R01MH108528, R01MH109885]
  5. FAPESP [2016/26189-0]
  6. Fundacao de Amparo a Pesquisa do Estado de Sao Paulo (FAPESP) [12/50880-4] Funding Source: FAPESP

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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.

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