Loss of glycosylation associated with the T183A mutation in human prion disease

Acta Neuropathol. 2004 Dec;108(6):476-84. doi: 10.1007/s00401-004-0913-4. Epub 2004 Oct 26.

Abstract

A heterozygous T183A mutation in the prion protein (PrP) gene, PRNP, was identified in a patient with histopathologically confirmed spongiform encephalopathy. Clinically, this form of prion disease was characterized by early-onset dementia as the predominant sign, along with global cerebral atrophy and hypometabolism. The age at onset was 40 years and the disease duration was 4 years. Additional neurological signs including cerebellar ataxia and EEG abnormalities were absent until late stages of the disease. The T183A mutation was not found in non-affected family members. This mutation results in the removal of one of the two consensus sites for glycosylation of PrP. Neuropathological examination revealed severe spongiform degeneration and neuronal loss in the neocortex, putamen and claustrum, small plaque-like PrP-immunoreactive deposits in the molecular layer of the cerebellum, and faint intracellular cytoplasmic PrP immunoreactivity. Western blot analysis of the patient's brain tissue showed protease K-resistant PrP with a definite preponderance of the monoglycosylated form. The additional appearance of a band representing diglycosylated PrPSc strongly suggests that non-mutated PrP also acquires protease resistance in the present setting. Cell culture experiments confirmed previous reports on intracellular retention of the mutant protein in vitro. This is the second report of a disease-causing T183A mutation of PrP, and the clinical, histological and genetic observations strongly suggest that T183A is a disease-causing mutation.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Blotting, Western
  • Brain / pathology*
  • Cell Line, Tumor
  • Creutzfeldt-Jakob Syndrome / genetics*
  • Creutzfeldt-Jakob Syndrome / pathology
  • Creutzfeldt-Jakob Syndrome / physiopathology
  • Glycosylation*
  • Humans
  • Immunohistochemistry
  • Male
  • Microscopy, Confocal
  • Mutation
  • Neuroblastoma
  • Pedigree
  • Polymerase Chain Reaction
  • PrPSc Proteins / genetics
  • Prion Diseases / genetics*
  • Prions / genetics*
  • Prions / metabolism

Substances

  • PrPSc Proteins
  • Prions