The genetics of prion diseases

Genet Med. 2010 Apr;12(4):187-95. doi: 10.1097/GIM.0b013e3181cd7374.

Abstract

Prion diseases are a rare group of fatal neurodegenerative disorders of humans and animals that manifest primarily as progressive dementia and ataxia. Unique to these diseases is the prion, a misfolded isoform of the prion protein that can transmit disease from cell to cell or host to host by associating with, and transforming, normal prion protein into the misfolded isoform (the pathogenic scrapie-inducing form). Although the majority of cases occur on a sporadic basis, and rarely result from exposure to prions, such as mad cow disease, 10-15% are attributable to the presence of an autosomal dominant mutation of the prion protein gene (PRNP). Single base pair changes, or the insertion of one or more multiples of a 24 base pair repeat segment, make up the known sequence alterations of PRNP associated with genetic prion disease. The common polymorphic codon 129 of PRNP also plays an important and complex role in risk and phenotype of sporadic and genetic prion disease. This review will focus on the clinical and histopathologic features of the genetic prion diseases. Selected mutations will be highlighted as a way to illustrate general phenotype-genotype correlations.

Publication types

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

MeSH terms

  • Brain / metabolism
  • Brain / pathology
  • Genetic Association Studies
  • Genetic Predisposition to Disease
  • Humans
  • Mutation*
  • Polymorphism, Genetic
  • Prion Diseases / classification
  • Prion Diseases / genetics*
  • Prion Diseases / pathology
  • Prion Proteins
  • Prions / genetics*

Substances

  • PRNP protein, human
  • Prion Proteins
  • Prions