Human stem cells as a model of motoneuron development and diseases

Ann N Y Acad Sci. 2010 Jun;1198:192-200. doi: 10.1111/j.1749-6632.2010.05537.x.


Human embryonic stem cells (hESCs) and human induced pluripotent stem cells (hiPSCs) possess the potential to become all cell and tissue types of the human body. Under chemically defined culture systems, hESCs and hiPSCs have been efficiently directed to functional spinal motoneurons and astrocytes. The differentiation process faithfully recapitulates the developmental process predicted from studies in vertebrate animals and human specimens, suggesting the usefulness of stem cell differentiation systems in understanding human cellular development. Motoneurons and astrocytes differentiated from genetically altered hESCs or disease hiPSCs exhibit predicted phenotypes. They thus offer a simplified dynamic model for analyzing pathological processes that lead to human motoneuron degeneration, which in turn may serve as a template for pharmaceutical screening. In addition, the human stem cell-derived motoneurons and astrocytes, including those specifically derived from a patient, may become a source for cell therapy.

Publication types

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

MeSH terms

  • Astrocytes / cytology
  • Astrocytes / physiology
  • Basic Helix-Loop-Helix Transcription Factors / genetics
  • Basic Helix-Loop-Helix Transcription Factors / physiology
  • Cell Differentiation
  • Drug Evaluation, Preclinical / methods
  • Embryonic Stem Cells / cytology*
  • Embryonic Stem Cells / physiology*
  • Hedgehog Proteins / physiology
  • Helix-Loop-Helix Motifs
  • Humans
  • Mitosis
  • Models, Neurological
  • Motor Neuron Disease / pathology
  • Motor Neuron Disease / physiopathology*
  • Motor Neurons / cytology*
  • Motor Neurons / physiology*
  • Nerve Degeneration
  • Nerve Tissue Proteins / genetics
  • Nerve Tissue Proteins / physiology
  • Oligodendrocyte Transcription Factor 2
  • Pluripotent Stem Cells / cytology*
  • Pluripotent Stem Cells / physiology*
  • Spinal Cord / embryology
  • Stem Cell Transplantation / methods


  • Basic Helix-Loop-Helix Transcription Factors
  • Hedgehog Proteins
  • Nerve Tissue Proteins
  • OLIG2 protein, human
  • Oligodendrocyte Transcription Factor 2
  • SHH protein, human