Glucose and weight control in mice with a designed ghrelin O-acyltransferase inhibitor

Science. 2010 Dec 17;330(6011):1689-92. doi: 10.1126/science.1196154. Epub 2010 Nov 18.

Abstract

Ghrelin is a gastric peptide hormone that stimulates weight gain in vertebrates. The biological activities of ghrelin require octanoylation of the peptide on Ser(3), an unusual posttranslational modification that is catalyzed by the enzyme ghrelin O-acyltransferase (GOAT). Here, we describe the design, synthesis, and characterization of GO-CoA-Tat, a peptide-based bisubstrate analog that antagonizes GOAT. GO-CoA-Tat potently inhibits GOAT in vitro, in cultured cells, and in mice. Intraperitoneal administration of GO-CoA-Tat improves glucose tolerance and reduces weight gain in wild-type mice but not in ghrelin-deficient mice, supporting the concept that its beneficial metabolic effects are due specifically to GOAT inhibition. In addition to serving as a research tool for mapping ghrelin actions, GO-CoA-Tat may help pave the way for clinical targeting of GOAT in metabolic diseases.

Publication types

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

MeSH terms

  • Acylation
  • Acyltransferases / antagonists & inhibitors*
  • Animals
  • Cell Survival / drug effects
  • Drug Design
  • Enzyme Inhibitors / chemical synthesis
  • Enzyme Inhibitors / pharmacology*
  • Enzyme Inhibitors / toxicity
  • Ghrelin / deficiency
  • Ghrelin / genetics
  • Ghrelin / metabolism*
  • Glucose / metabolism*
  • Glucose Tolerance Test
  • HeLa Cells
  • Homeostasis
  • Humans
  • Insulin / metabolism
  • Ion Channels / metabolism
  • Islets of Langerhans / drug effects
  • Islets of Langerhans / metabolism
  • Male
  • Membrane Proteins
  • Mice
  • Mice, Inbred C57BL
  • Mice, Knockout
  • Mitochondrial Proteins / metabolism
  • Peptides / chemical synthesis
  • Peptides / pharmacology*
  • Peptides / toxicity
  • Uncoupling Protein 2
  • Weight Gain / drug effects*

Substances

  • Enzyme Inhibitors
  • GO-CoA-Tat
  • Ghrelin
  • Insulin
  • Ion Channels
  • Membrane Proteins
  • Mitochondrial Proteins
  • Peptides
  • Uncoupling Protein 2
  • Acyltransferases
  • Mboat4 protein, mouse
  • Glucose