CALCB splice region pathogenic variants leading to plasma cell neurotropic enrichment in type 1 autoimmune pancreatitis

Cell Death Dis. 2017 Feb 2;8(2):e2591. doi: 10.1038/cddis.2017.32.

Abstract

Recently, we have demonstrated that PRSS1 mutations cause ectopic trypsinogen activation and thereby result in type 1 autoimmune pancreatitis (AIP). However, the molecules involved in inducing obliterative vasculitis and perineural inflammation in the pancreas are not well-described. The present study applied whole-exome sequencing (WES) to determine the underlying etiology and revealed novel missense splice region variants, CALCB c.88T>C (p.Ser30Pro) and IR [1]-mutants, in 2 of the 3 families and 2 of 26 unrelated patients with type 1 AIP. In vitro, both of the mutants displayed decreased βCGRP, ERK1/2 phosphorylation, and co-localized with endoplasmic reticulum and Golgi apparatus. The novel pathogenic variant identified in this case should contribute to our understanding of the expanding spectrum of AIP.

MeSH terms

  • Autoimmune Diseases / genetics*
  • Autoimmune Diseases / pathology*
  • Calcitonin Gene-Related Peptide / genetics*
  • Cell Line
  • Endoplasmic Reticulum / genetics
  • Female
  • Golgi Apparatus / genetics
  • HEK293 Cells
  • Humans
  • Inflammation / genetics
  • Inflammation / pathology
  • MAP Kinase Signaling System / genetics
  • Male
  • Mutation / genetics*
  • Pancreas / pathology
  • Pancreatitis / genetics*
  • Pancreatitis / pathology*
  • Phosphorylation / genetics
  • Plasma Cells / pathology*
  • Trypsin / genetics

Substances

  • Trypsin
  • Calcitonin Gene-Related Peptide