Protocol

Protein Misfolding and Disease

Volume 232 of the series Methods in Molecular Biology™ pp 231-243

Application of Chemical Chaperones to the Rescue of Folding Defects

  • Tip W. LooAffiliated withDepartments of Medicine and Biochemistry, University of Toronto
  • , David M. ClarkeAffiliated withDepartments of Medicine and Biochemistry, University of Toronto

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Abstract

Abnormal protein folding in the endoplasmic reticulum (ER) is the cause of more than 40 diseases such as cystic fibrosis (CF), familial hypercholesterolemia, long QT syndrome, diabetes insipidus, and Alzheimer's disease (AD) (reviewed in refs. 1,2). For diseases caused by protein misfolding, an important goal would be to develop drugs or small molecules to prevent misfolding.