Second-Generation Pharmacological Chaperones: Beyond Inhibitors

Protein misfolding induced by missense mutations is the source of hundreds of conformational diseases. The cell quality control may eliminate nascent misfolded proteins, such as enzymes, and a pathological loss-of-function may result from their early degradation. Since the proof of concept in the 20...

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Veröffentlicht in:	Molecules (Basel, Switzerland) Switzerland), 2020-07, Vol.25 (14), p.3145
Hauptverfasser:	Tran, My Lan, Génisson, Yves, Ballereau, Stéphanie, Dehoux, Cécile
Format:	Artikel
Sprache:	eng
Schlagworte:	allosteric ligand Bibliographic data bases Binding Business competition Chaperones Chemical Sciences Competition conformational disease Disease Drug development Enzymatic activity Enzyme Activators - chemistry Enzyme Activators - therapeutic use Enzyme activity Enzyme Inhibitors - chemistry Enzyme Inhibitors - therapeutic use Enzymes Genetic disorders Humans Inhibitors Ligands lysosomal storage disease Metabolic disorders Metabolism Metabolites Missense mutation Molecular Chaperones - chemistry Molecular Chaperones - therapeutic use Mutation non-inhibitory chaperones Organic chemistry pharmacological chaperones Pharmacology Protein Folding Proteins Quality control Review
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