Pharmacological Chaperones in Protein Aggregation Disorders

Chaperones are molecules that assist in the proper folding of nascent polypeptide chain and prevent protein from aggregation. There are at least three types of chaperones: molecular chaperones, chemical chaperones and pharmacological chaperones. Unlike molecular chaperones that are enzymes or proteins, chemical chaperones are small molecular weight organic compounds. Molecular chaperones catalyze a folding process, while chemical chaperones simply assist in the protein folding process by making the malfolded or unstable protein stay away from aggregation. On the other hand, pharmacological chaperones are a class of molecules that bind and inhibit the unstable or misfolded proteins. Therefore, they act specific for a particular protein. Various studies have yielded insights that these pharmacological chaperones would be effective for a large number of diseases including Alzheimer’s disease, nephrogenic diabetes insipidus, cystic fibrosis, lysosomal storage diseases, phenylketonuria, methylmalonic aciduria, etc. In the present chapter, we have described various diseases for which the use of pharmacological chaperones has been proved successful.