Therapeutic approaches to protein-misfolding diseases

Nature. 2003 Dec 18;426(6968):905-9. doi: 10.1038/nature02265.


Several sporadic and genetic diseases are caused by protein misfolding. These include cystic fibrosis and other devastating diseases of childhood as well as Alzheimer's, Parkinson's and other debilitating maladies of the elderly. A unified view of the molecular and cellular pathogenesis of these conditions has led to the search for chemical chaperones that can slow, arrest or revert disease progression. Molecules are now emerging that link our biophysical insights with our therapeutic aspirations.

Publication types

  • Review

MeSH terms

  • Alzheimer Disease / metabolism
  • Alzheimer Disease / therapy*
  • Animals
  • Humans
  • Molecular Chaperones / metabolism
  • Protein Denaturation
  • Protein Folding*
  • Proteins / chemistry*
  • Proteins / metabolism*
  • Thermodynamics


  • Molecular Chaperones
  • Proteins