Lupus-associated causal mutation in neutrophil cytosolic factor 2 (NCF2) brings unique insights to the structure and function of NADPH oxidase

Proc Natl Acad Sci U S A. 2012 Jan 10;109(2):E59-67. doi: 10.1073/pnas.1113251108. Epub 2011 Dec 27.

Abstract

Systemic lupus erythematosus (SLE), the prototypic systemic autoimmune disease, is a debilitating multisystem autoimmune disorder characterized by chronic inflammation and extensive immune dysregulation in multiple organ systems, resulting in significant morbidity and mortality. Here, we present a multidisciplinary approach resulting in the identification of neutrophil cytosolic factor 2 (NCF2) as an important risk factor for SLE and the detailed characterization of its causal variant. We show that NCF2 is strongly associated with increased SLE risk in two independent populations: childhood-onset SLE and adult-onset SLE. The association between NCF2 and SLE can be attributed to a single nonsynonymous coding mutation in exon 12, the effect of which is the substitution of histidine-389 with glutamine (H389Q) in the PB1 domain of the NCF2 protein, with glutamine being the risk allele. Computational modeling suggests that the NCF2 H389Q mutation reduces the binding efficiency of NCF2 with the guanine nucleotide exchange factor Vav1. The model predicts that NCF2/H389 residue interacts with Vav1 residues E509, N510, E556, and G559 in the ZF domain of Vav1. Furthermore, replacing H389 with Q results in 1.5 kcal/mol weaker binding. To examine the effect of the NCF2 H389Q mutation on NADPH oxidase function, site-specific mutations at the 389 position in NCF2 were tested. Results show that an H389Q mutation causes a twofold decrease in reactive oxygen species production induced by the activation of the Vav-dependent Fcγ receptor-elicited NADPH oxidase activity. Our study completes the chain of evidence from genetic association to specific molecular function.

Publication types

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

MeSH terms

  • Amino Acid Sequence
  • California
  • Genetic Predisposition to Disease / genetics*
  • Genetic Variation*
  • Genotype
  • Humans
  • Lupus Erythematosus, Systemic / genetics*
  • Models, Molecular*
  • Molecular Sequence Data
  • Multiprotein Complexes / chemistry
  • Multiprotein Complexes / genetics*
  • Mutation, Missense / genetics
  • NADPH Oxidases / chemistry
  • NADPH Oxidases / genetics
  • NADPH Oxidases / metabolism*
  • Plasmids / genetics
  • Polymorphism, Single Nucleotide / genetics
  • Principal Component Analysis
  • Protein Binding
  • Proto-Oncogene Proteins c-vav / chemistry
  • Proto-Oncogene Proteins c-vav / metabolism
  • Reactive Oxygen Species / metabolism
  • rac1 GTP-Binding Protein / chemistry

Substances

  • Multiprotein Complexes
  • Proto-Oncogene Proteins c-vav
  • RAC1 protein, human
  • Reactive Oxygen Species
  • VAV1 protein, human
  • NADPH Oxidases
  • NCF2 protein, human
  • NCF4 protein, human
  • rac1 GTP-Binding Protein