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Using ASMase knockout mice to model human diseases

Guoqiang Hua1, Richard Kolesnick

  • 1Memorial Sloan-Kettering Cancer Center, New York, NY 10065, USA.

Handbook of Experimental Pharmacology
|April 9, 2013
PubMed

Insights

Acid sphingomyelinase (ASMase) initiates sphingomyelin/ceramide signaling, crucial for stress responses. This pathway regulates tissue injury across multiple organs, offering therapeutic intervention targets.

Area of Science:

  • Biochemistry
  • Cellular Biology
  • Physiology

Background:

  • Acid sphingomyelinase (ASMase) is a critical enzyme in sphingomyelin/ceramide signaling.
  • This pathway is activated by various stress stimuli and implicated in numerous diseases.
  • ASMase knockout mice have been instrumental in understanding its clinical relevance.

Purpose of the Study:

  • To review the evolutionary conservation of sphingolipid stress signaling.
  • To highlight mammalian adaptations in organotypic responses.
  • To underscore the role of ceramide generation in organ injury and fate determination.

Main Methods:

  • Utilizing data from ASMase knockout mouse models.
  • Analyzing evolutionary conservation of sphingolipid pathways.
  • Reviewing literature on ceramide generation in disease states.

Main Results:

  • Ceramide generation by ASMase governs injury in diverse organs including tumor, gut, ovary, brain, lung, heart, and liver.
  • The pathway is evolutionarily conserved, with mammalian adaptations enabling organ-specific responses.
  • ASMase acts as a molecular switch determining tissue injury extent and organ fate.

Conclusions:

  • Sphingomyelin/ceramide signaling, initiated by ASMase, is a fundamental stress response pathway.
  • Understanding this pathway's role in organ injury is vital for therapeutic development.
  • Targeting ASMase offers potential for new pharmacologic interventions in various diseases.

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