Pharmacological Chaperone Therapy

Benito, J.M., Garcia Fernandez, J.M., and Ortiz Mellet, C. (2011) Pharmacological chaperone therapy for Gaucher disease a patent review. Expert Opin. Iher. Pat, 21, 885 -903. 

Substrate reduction therapy (SRT) involves the inhibition of substrate synthesis to a level where the load falls within the capacity of the mutant enzyme that exhibits residual function (Platt and Jeyakumar, 2008). Thus this approach, as in the case of pharmacological chaperones, may be dependent on the type of mutation responsible for disease in an individual. 

A number of different pharmacological chaperones that correct folding states of mutant proteins and enable their trafficking to the proper locale would be excellent candidates for a new molecular therapy of human genetic disorders. It can be expected that orally administered pharmacological chaperones will become the standard treatment for broad genetic disorders with protein misfolding. 

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