Structure and inhibition mechanism of the catalytic domain of human squalene epoxidase

Nat Commun. 2019 Jan 9;10(1):97. doi: 10.1038/s41467-018-07928-x.

Abstract

Squalene epoxidase (SQLE), also known as squalene monooxygenase, catalyzes the stereospecific conversion of squalene to 2,3(S)-oxidosqualene, a key step in cholesterol biosynthesis. SQLE inhibition is targeted for the treatment of hypercholesteremia, cancer, and fungal infections. However, lack of structure-function understanding has hindered further progression of its inhibitors. We have determined the first three-dimensional high-resolution crystal structures of human SQLE catalytic domain with small molecule inhibitors (2.3 Å and 2.5 Å). Comparison with its unliganded state (3.0 Å) reveals conformational rearrangements upon inhibitor binding, thus allowing deeper interpretation of known structure-activity relationships. We use the human SQLE structure to further understand the specificity of terbinafine, an approved agent targeting fungal SQLE, and to provide the structural insights into terbinafine-resistant mutants encountered in the clinic. Collectively, these findings elucidate the structural basis for the specificity of the epoxidation reaction catalyzed by SQLE and enable further rational development of next-generation inhibitors.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Catalytic Domain
  • Cell Line
  • Gene Expression Regulation, Enzymologic / drug effects
  • Humans
  • Insecta
  • Protein Conformation
  • Protein Domains
  • Squalene / metabolism
  • Squalene Monooxygenase / antagonists & inhibitors
  • Squalene Monooxygenase / chemistry*
  • Squalene Monooxygenase / metabolism*

Substances

  • Squalene
  • Squalene Monooxygenase