Presenilin-1 and -2 are molecular targets for gamma-secretase inhibitors

J Biol Chem. 2000 Nov 3;275(44):34086-91. doi: 10.1074/jbc.M005430200.

Abstract

Presenilins are integral membrane protein involved in the production of amyloid beta-protein. Mutations of the presenilin-1 and -2 gene are associated with familial Alzheimer's disease and are thought to alter gamma-secretase cleavage of the beta-amyloid precursor protein, leading to increased production of longer and more amyloidogenic forms of A beta, the 4-kDa beta-peptide. Here, we show that radiolabeled gamma-secretase inhibitors bind to mammalian cell membranes, and a benzophenone analog specifically photocross-links three major membrane polypeptides. A positive correlation is observed among these compounds for inhibition of cellular A beta formation, inhibition of membrane binding and cross-linking. Immunological techniques establish N- and C-terminal fragments of presenilin-1 as specifically cross-linked polypeptides. Furthermore, binding of gamma-secretase inhibitors to embryonic membranes derived from presenilin-1 knockout embryos is reduced in a gene dose-dependent manner. In addition, C-terminal fragments of presenilin-2 are specifically cross-linked. Taken together, these results indicate that potent and selective gamma-secretase inhibitors block A beta formation by binding to presenilin-1 and -2.

MeSH terms

  • Amyloid Precursor Protein Secretases
  • Cell Membrane / metabolism
  • Endopeptidases / drug effects*
  • Endopeptidases / metabolism
  • Enzyme Inhibitors / metabolism*
  • Membrane Proteins / metabolism*
  • Precipitin Tests
  • Presenilin-1
  • Presenilin-2
  • Substrate Specificity

Substances

  • Enzyme Inhibitors
  • Membrane Proteins
  • Presenilin-1
  • Presenilin-2
  • Amyloid Precursor Protein Secretases
  • Endopeptidases