Chemical modulators of autophagy as biological probes and potential therapeutics

Nat Chem Biol. 2011 Jan;7(1):9-17. doi: 10.1038/nchembio.500.


Autophagy is an evolutionarily conserved mechanism for protein degradation that is critical for the maintenance of homeostasis in man. Autophagy has unexpected pleiotropic functions that favor survival of the cell, including nutrient supply under starvation, cleaning of the cellular interior, defense against infection and antigen presentation. Moreover, defective autophagy is associated with a diverse range of disease states, including neurodegeneration, cancer and Crohn's disease. Here we discuss the roles of mammalian autophagy in health and disease and highlight recent advances in pharmacological manipulation of autophagic pathways as a therapeutic strategy for a variety of pathological conditions.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Autophagy* / drug effects
  • Crohn Disease / drug therapy*
  • Crohn Disease / metabolism
  • Crohn Disease / pathology
  • Drug Discovery / methods*
  • Humans
  • Lysosome-Associated Membrane Glycoproteins / metabolism
  • Lysosomes / metabolism
  • Neoplasms / drug therapy*
  • Neoplasms / metabolism
  • Neoplasms / pathology
  • Nerve Degeneration / drug therapy*
  • Nerve Degeneration / metabolism
  • Nerve Degeneration / pathology


  • Lysosome-Associated Membrane Glycoproteins