Transgenesis applied to transmissible spongiform encephalopathies

Transgenic Res. 2002 Dec;11(6):547-64. doi: 10.1023/a:1021125510220.

Abstract

Transmissible spongiform encephalopathies (TSE) are fatal neurodegenerative disorders present in various mammals. TSEs have been studies intensively, even more so following the BSE crisis and the subsequent threat of a human nvCJD epidemic. In the 'protein-only' hypothesis, the infectious agent, called prion, is assumed to be a misfolded host protein. Transgenesis has mainly been applied to study the role of this protein, its structure-function relationship with respect to its pathogenic properties and to assess the genetic origin of the well-recognised species barrier effect. This approach has somewhat supplemented the lack of in vitro models. This review will try to summarise the impressive work that has been done in this field. Although many questions remain unanswered, transgenic experiments have and will still improve our knowledge on this disease and might help us to develop critically needed therapeutic approaches.

Publication types

  • Review

MeSH terms

  • Animals
  • Disease Models, Animal
  • GPI-Linked Proteins
  • Humans
  • Mutation
  • Prion Diseases / etiology
  • Prion Diseases / genetics*
  • Prion Diseases / transmission
  • Prions / genetics
  • Prions / physiology
  • Species Specificity
  • Transgenes / physiology*

Substances

  • GPI-Linked Proteins
  • PRND protein, human
  • Prions