COPA mutations impair ER-Golgi transport and cause hereditary autoimmune-mediated lung disease and arthritis

Nat Genet. 2015 Jun;47(6):654-60. doi: 10.1038/ng.3279. Epub 2015 Apr 20.

Abstract

Unbiased genetic studies have uncovered surprising molecular mechanisms in human cellular immunity and autoimmunity. We performed whole-exome sequencing and targeted sequencing in five families with an apparent mendelian syndrome of autoimmunity characterized by high-titer autoantibodies, inflammatory arthritis and interstitial lung disease. We identified four unique deleterious variants in the COPA gene (encoding coatomer subunit α) affecting the same functional domain. Hypothesizing that mutant COPA leads to defective intracellular transport via coat protein complex I (COPI), we show that COPA variants impair binding to proteins targeted for retrograde Golgi-to-ER transport. Additionally, expression of mutant COPA results in ER stress and the upregulation of cytokines priming for a T helper type 17 (TH17) response. Patient-derived CD4(+) T cells also demonstrate significant skewing toward a TH17 phenotype that is implicated in autoimmunity. Our findings uncover an unexpected molecular link between a vesicular transport protein and a syndrome of autoimmunity manifested by lung and joint disease.

Publication types

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

MeSH terms

  • Amino Acid Sequence
  • Arthritis / genetics*
  • Autoimmune Diseases / genetics*
  • Child, Preschool
  • Coatomer Protein / genetics*
  • Endoplasmic Reticulum / metabolism
  • Endoplasmic Reticulum Stress
  • Female
  • Genetic Association Studies
  • Genetic Predisposition to Disease
  • Golgi Apparatus / metabolism*
  • HEK293 Cells
  • Humans
  • Infant
  • Lod Score
  • Lung Diseases, Interstitial / genetics*
  • Male
  • Molecular Sequence Data
  • Pedigree
  • Protein Transport

Substances

  • Coatomer Protein