Mutations of the anti-mullerian hormone gene in patients with persistent mullerian duct syndrome: biosynthesis, secretion, and processing of the abnormal proteins and analysis using a three-dimensional model

Mol Endocrinol. 2004 Mar;18(3):708-21. doi: 10.1210/me.2003-0358. Epub 2003 Dec 12.

Abstract

Anti-Müllerian hormone (AMH), a TGF-beta family member, determines whether an individual develops a uterus and Fallopian tubes. Mutations in the AMH gene lead to persistent Müllerian duct syndrome in males. The wild-type human AMH protein is synthesized as a disulfide-linked dimer of two identical 70-kDa polypeptides, which undergoes proteolytic processing to generate a 110-kDa N-terminal dimer and a bioactive 25-kDa TGF-beta-like C-terminal dimer. We have studied the biosynthesis and secretion of wild-type AMH and of seven persistent Müllerian duct syndrome proteins, containing mutations in either the N- or C-terminal domain. Mutant proteins lacking the C-terminal domain are secreted more rapidly than full-length AMH, whereas single amino acid changes in both domains can have profound effects on protein stability and folding. The addition of a cysteine in an N-terminal domain mutant, R194C, prevents proper folding, whereas the elimination of the cysteine involved in forming the interchain disulfide bond, in a C-terminal domain mutant, C525Y, leads to a truncation at the C terminus. A molecular model of the AMH C-terminal domain provides insights into how some mutations could affect biosynthesis and function.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Amino Acid Sequence
  • Animals
  • Anti-Mullerian Hormone
  • COS Cells
  • Cricetinae
  • Cysteine / genetics
  • Disorders of Sex Development / genetics*
  • Glycoproteins / chemistry*
  • Glycoproteins / genetics*
  • Glycoproteins / metabolism*
  • Glycosylation
  • Humans
  • Models, Molecular
  • Molecular Sequence Data
  • Mutation*
  • Protein Conformation
  • Protein Folding
  • Protein Processing, Post-Translational
  • Protein Structure, Tertiary
  • Testicular Hormones / chemistry*
  • Testicular Hormones / genetics*
  • Testicular Hormones / metabolism*

Substances

  • Glycoproteins
  • Testicular Hormones
  • Anti-Mullerian Hormone
  • Cysteine