High-Content Small Molecule Screening Strategies for Stem-Cell-Derived Motor Neurons

Eunju Pack-Chung, Courtney A. Ackeifi, Lida Katsimpardi, Kevin J. Kim, Maureen Sherry Lynes, Nina R. Makhortova, Lee L. Rubin

Research output: Chapter in Book/Report/Conference proceedingChapterpeer-review


Human pluripotent stem cells possess great potential as a source of large numbers of defined cell populations that can be used for therapeutic applications and for pharmaceutical screening. By using disease-specific stem cells, in vitro disease models can be established that better recapitulate human diseases and lead to the development of more effective therapeutics and more informative disease mechanism studies. We have developed a high-content screening (HCS) assay using embryonic-stem-cell-derived motor neurons to study disease-related processes such as differentiation, survival, and protein expression. In particular, the assay described here is aimed at finding small molecules that can modulate the level of a key protein—survival of motor neuron—implicated in the human motor neuron disease—spinal muscular atrophy. A detailed protocol for the assay setups, including the parameters for growth conditions, compound library selection, and hit validation methods, are described.

Original languageEnglish
Title of host publicationMethods in Cell Biology
PublisherAcademic Press Inc.
Number of pages21
StatePublished - Jan 2012
Externally publishedYes

Publication series

NameMethods in Cell Biology
ISSN (Print)0091-679X


  • Cytoplasm
  • Elevation of Smn expression
  • High content imaging
  • Immunostaining
  • Lineage specification
  • Motor neuron differentiation
  • Motor neurons
  • Mouse
  • Neurodegenerative diseases
  • Neuronal differentiation
  • Nucleus
  • Small-molecule screen
  • Sonic hedgehog
  • Spinal muscular atrophy
  • Stem cell culture
  • Survival of motor neuron protein


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