Unifying perspectives on the activity and genotypic targeting of pharmacological chaperones.

Abstract excerpt

Several diseases of protein misfolding can now be treated with an emerging class of therapeutics known as pharmacological chaperones, pharmacochaperones, or correctors. These small molecules exploit the universal thermodynamic coupling between ligand binding and protein folding to suppress conformational defects that disrupt protein homeostasis. While the mechanistic basis of their activity is quite simple in...

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