Protein misfolding and disease; protein refolding and therapy

FEBS Lett. 2001 Jun 8;498(2-3):204-7. doi: 10.1016/s0014-5793(01)02486-3.


Diverse human disorders, including several neurodegenerative diseases and systemic amyloidosis, are thought to arise from the misfolding and aggregation of an underlying protein. Recent findings strongly support this hypothesis and have increased our understanding of the molecular mechanism of protein conformational disorders. Many questions are still pending, but the data overall suggest that correction of protein misfolding constitutes a viable therapeutic strategy for conformational diseases.

Publication types

  • Review

MeSH terms

  • Animals
  • Central Nervous System Diseases / genetics
  • Central Nervous System Diseases / metabolism*
  • Central Nervous System Diseases / therapy
  • Humans
  • Peptides / therapeutic use
  • Protein Conformation*
  • Protein Folding*
  • Proteins / chemistry
  • Proteins / genetics
  • Proteins / metabolism*
  • Structure-Activity Relationship


  • Peptides
  • Proteins