Discovery of a Small-Molecule Probe for V-ATPase Function

J Am Chem Soc. 2015 Apr 29;137(16):5563-8. doi: 10.1021/jacs.5b02150. Epub 2015 Apr 20.

Abstract

Lysosomes perform a critical cellular function as a site of degradation for diverse cargoes including proteins, organelles, and pathogens delivered through distinct pathways, and defects in lysosomal function have been implicated in a number of diseases. Recent studies have elucidated roles for the lysosome in the regulation of protein synthesis, metabolism, membrane integrity, and other processes involved in homeostasis. Complex small-molecule natural products have greatly contributed to the investigation of lysosomal function in cellular physiology. Here we report the discovery of a novel, small-molecule modulator of lysosomal acidification derived from diversity-oriented synthesis through high-content screening.

Publication types

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

MeSH terms

  • Cell Line, Tumor
  • Cell Survival / drug effects
  • Enzyme Inhibitors / chemistry*
  • Enzyme Inhibitors / pharmacology*
  • Humans
  • Lysosomes / drug effects*
  • Lysosomes / enzymology*
  • Lysosomes / metabolism
  • Macrolides / pharmacology
  • Small Molecule Libraries / chemistry*
  • Small Molecule Libraries / pharmacology*
  • Vacuolar Proton-Translocating ATPases / antagonists & inhibitors
  • Vacuolar Proton-Translocating ATPases / metabolism*

Substances

  • Enzyme Inhibitors
  • Macrolides
  • Small Molecule Libraries
  • bafilomycin A1
  • Vacuolar Proton-Translocating ATPases