Using pharmacological chaperones to restore proteostasis

摘要: Normal organismal physiology depends on the maintenance of proteostasis in each cellular compartment to achieve a delicate balance between protein synthesis, folding, trafficking, and degradation while minimizing misfolding aggregation. Defective leads numerous diseases. Pharmacological chaperones are cell-permeant small molecules that promote proper folding trafficking via direct binding protein. They stabilize their target protein-pharmacological chaperone state, increasing natively folded population can effectively engage machinery for transport final destination function. Here, as regards application pharmacological chaperones, we focus capability lysosomal enzymes, G coupled receptors (GPCRs), ion channels, which is presently an important drug target. hold great promise potential therapeutics ameliorate variety