A small molecule that binds Hedgehog and blocks its signaling in human cells

Nat Chem Biol. 2009 Mar;5(3):154-6. doi: 10.1038/nchembio.142. Epub 2009 Jan 18.

Abstract

Small-molecule inhibition of extracellular proteins that activate membrane receptors has proven to be extremely challenging. Diversity-oriented synthesis and small-molecule microarrays enabled the discovery of robotnikinin, a small molecule that binds the extracellular Sonic hedgehog (Shh) protein and blocks Shh signaling in cell lines, human primary keratinocytes and a synthetic model of human skin. Shh pathway activity is rescued by small-molecule agonists of Smoothened, which functions immediately downstream of the Shh receptor Patched.

Publication types

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

MeSH terms

  • 3T3 Cells
  • Animals
  • Drug Discovery
  • Hedgehog Proteins / metabolism*
  • Humans
  • Keratinocytes / drug effects
  • Keratinocytes / metabolism
  • Lactams / metabolism
  • Lactams / pharmacology*
  • Lactones / metabolism
  • Lactones / pharmacology*
  • Mice
  • Patched Receptors
  • Receptors, Cell Surface / genetics
  • Receptors, Cell Surface / metabolism
  • Signal Transduction / drug effects*

Substances

  • Hedgehog Proteins
  • Lactams
  • Lactones
  • Patched Receptors
  • Receptors, Cell Surface
  • robotnikinin

Associated data

  • PubChem-Substance/56431959
  • PubChem-Substance/56431960
  • PubChem-Substance/56431961
  • PubChem-Substance/56431962
  • PubChem-Substance/56431963
  • PubChem-Substance/56431964
  • PubChem-Substance/56431965
  • PubChem-Substance/56431966
  • PubChem-Substance/56431967
  • PubChem-Substance/56431968
  • PubChem-Substance/56431969
  • PubChem-Substance/56431970
  • PubChem-Substance/56431971
  • PubChem-Substance/56431972
  • PubChem-Substance/56431973
  • PubChem-Substance/56431974
  • PubChem-Substance/56431975
  • PubChem-Substance/56431976
  • PubChem-Substance/56431977
  • PubChem-Substance/56431978
  • PubChem-Substance/56431979
  • PubChem-Substance/56431980