High-throughput screening assay for the identification of compounds regulating self-renewal and differentiation in human embryonic stem cells

Cell Stem Cell. 2008 Jun 5;2(6):602-12. doi: 10.1016/j.stem.2008.05.010.

Abstract

High-throughput screening (HTS) of chemical libraries has become a critical tool in basic biology and drug discovery. However, its implementation and the adaptation of high-content assays to human embryonic stem cells (hESCs) have been hampered by multiple technical challenges. Here we present a strategy to adapt hESCs to HTS conditions, resulting in an assay suitable for the discovery of small molecules that drive hESC self-renewal or differentiation. Use of this new assay has led to the identification of several marketed drugs and natural compounds promoting short-term hESC maintenance and compounds directing early lineage choice during differentiation. Global gene expression analysis upon drug treatment defines known and novel pathways correlated to hESC self-renewal and differentiation. Our results demonstrate feasibility of hESC-based HTS and enhance the repertoire of chemical compounds for manipulating hESC fate. The availability of high-content assays should accelerate progress in basic and translational hESC biology.

Publication types

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

MeSH terms

  • Animals
  • Biological Assay*
  • Cell Differentiation / drug effects
  • Dose-Response Relationship, Drug
  • Drug Design
  • Drug Evaluation, Preclinical
  • Embryo Research*
  • Embryonic Stem Cells / cytology*
  • Embryonic Stem Cells / drug effects*
  • Humans
  • Mice
  • Microarray Analysis
  • Regeneration / drug effects
  • Research Design / trends*
  • Small Molecule Libraries / pharmacology*
  • Small Molecule Libraries / therapeutic use

Substances

  • Small Molecule Libraries

Associated data

  • GEO/GSE11302