Patient-Specific Induced Pluripotent Stem Cells for Disease Modeling and Phenotypic Drug Discovery

J Med Chem. 2016 Jan 14;59(1):2-15. doi: 10.1021/acs.jmedchem.5b00789. Epub 2015 Sep 4.

Abstract

In vitro cell models are invaluable tools for studying diseases and discovering drugs. Human induced pluripotent stem cells, particularly derived from patients, are an advantageous resource for generating ample supplies of cells to create unique platforms that model disease. This manuscript will review recent developments in modeling a variety of diseases (including their cellular phenotypes) with induced pluripotent stem cells derived from patients. It will also describe how researchers have exploited these models to validate drugs as potential therapeutics for these devastating diseases.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Drug Discovery / methods*
  • Humans
  • Induced Pluripotent Stem Cells*
  • Models, Biological
  • Phenotype
  • Precision Medicine*
  • Stem Cell Transplantation / methods*