Interstitial lung disease in a baby with a de novo mutation in the SFTPC gene

Eur Respir J. 2004 Jul;24(1):30-9. doi: 10.1183/09031936.04.00000104.

Abstract

Mutations in the surfactant protein C gene (SFTPC) were recently reported in patients with interstitial lung disease. In a 13-month-old infant with severe respiratory insufficiency, a lung biopsy elicited combined histological patterns of nonspecific interstitial pneumonia and pulmonary alveolar proteinosis. Immunohistochemical and biochemical analyses showed an intra-alveolar accumulation of surfactant protein (SP)-A, precursors of SP-B, mature SP-B, aberrantly processed proSP-C, as well as mono- and dimeric SP-C. Sequencing of genomic DNA detected a de novo heterozygous missense mutation of the SFTPC gene (g.1286T>C) resulting in a substitution of threonine for isoleucine (173T) in the C-terminal propeptide. At the ultrastructural level, abnormal transport vesicles were detected in type-II pneumocytes. Fusion proteins, consisting of enhanced green fluorescent protein and wild-type or mutant proSP-C, were used to evaluate protein trafficking in vitro. In contrast to wild-type proSP-C, mutant proSP-C was routed to early endosomes when transfected into A549 epithelial cells. In contrast to previously reported mutations, the 173T represents a new class of surfactant protein C gene mutations, which is marked by a distinct trafficking, processing, palmitoylation, and secretion of the mutant and wild-type surfactant protein C. This report heralds the emerging diversity of phenotypes associated with the expression of mutant surfactant C proteins.

Publication types

  • Case Reports
  • Comparative Study
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.
  • Review

MeSH terms

  • Base Sequence
  • Biopsy, Needle
  • Blotting, Western
  • Genetic Predisposition to Disease*
  • Genetic Testing
  • Humans
  • Immunohistochemistry
  • Infant, Newborn
  • Lung Diseases, Interstitial / genetics*
  • Lung Diseases, Interstitial / pathology*
  • Male
  • Molecular Sequence Data
  • Mutation, Missense*
  • Polymerase Chain Reaction
  • Prognosis
  • Protein C / genetics*
  • Sensitivity and Specificity

Substances

  • Protein C