Assay strategies for identification of therapeutic leads that target protein trafficking

摘要: Receptors, enzymes, and ion channels are traditional targets of therapeutic development. A common strategy is to target these proteins with agents that either activate or suppress their activity ligands substrates occupy orthosteric sites have allosteric interactions. An alternative approach involves regulation protein trafficking. In principle, this enables 'rescue' misfolded misrouted mutant restore function, 'shipwrecking' undesirable by targeting them for destruction, levels partially expressed wild type (WT) at functional action. Here, we present drug discovery strategies identify 'pharmacoperones', which small molecules serve as molecular templates cause otherwise fold route correctly.