Modeling Motor Neuron Resilience in ALS Using Stem Cells

Stem Cell Reports. 2019 Jun 11;12(6):1329-1341. doi: 10.1016/j.stemcr.2019.04.009. Epub 2019 May 9.

Abstract

Oculomotor neurons, which regulate eye movement, are resilient to degeneration in the lethal motor neuron disease amyotrophic lateral sclerosis (ALS). It would be highly advantageous if motor neuron resilience could be modeled in vitro. Toward this goal, we generated a high proportion of oculomotor neurons from mouse embryonic stem cells through temporal overexpression of PHOX2A in neuronal progenitors. We demonstrate, using electrophysiology, immunocytochemistry, and RNA sequencing, that in vitro-generated neurons are bona fide oculomotor neurons based on their cellular properties and similarity to their in vivo counterpart in rodent and man. We also show that in vitro-generated oculomotor neurons display a robust activation of survival-promoting Akt signaling and are more resilient to the ALS-like toxicity of kainic acid than spinal motor neurons. Thus, we can generate bona fide oculomotor neurons in vitro that display a resilience similar to that seen in vivo.

Keywords: ALS; LCM sequencing; Onuf’s nucleus; Phox2a; RNA sequencing; amyotrophic lateral sclerosis; neuronal vulnerability and resistance; oculomotor neurons; spinal motor neurons; stem cells.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Amyotrophic Lateral Sclerosis / metabolism*
  • Amyotrophic Lateral Sclerosis / pathology
  • Animals
  • Cell Differentiation*
  • Cell Survival
  • Homeodomain Proteins / metabolism
  • Humans
  • Mice
  • Motor Neurons / metabolism*
  • Motor Neurons / pathology
  • Mouse Embryonic Stem Cells / metabolism*
  • Mouse Embryonic Stem Cells / pathology
  • Proto-Oncogene Proteins c-akt / metabolism
  • Signal Transduction*

Substances

  • Homeodomain Proteins
  • PHOX2A protein, human
  • Phox2a protein, mouse
  • Proto-Oncogene Proteins c-akt