Pharmacological Chaperones and Protein Conformational Diseases: Approaches of Computational Structural Biology.

ABSTRACT: Whenever a protein fails to fold into its native structure, a profound detrimental effect is likely to occur, and a disease is often developed. Protein conformational disorders arise when proteins adopt abnormal conformations due to a pathological gene variant that turns into gain/loss of function or improper localization/degradation. Pharmacological chaperones are small molecules restoring the correct folding of a protein suitable for treating conformational diseases. Small molecules like these bind poorly folded proteins similarly to physiological chaperones, bridging non-covalent interactions (hydrogen bonds, electrostatic interactions, and van der Waals contacts) loosened or lost due to mutations. Pharmacological chaperone development involves, among other things, structural biology in

SUBMITTER: Grasso D 

PROVIDER: S-EPMC10054308 | biostudies-literature | 2023 Mar

REPOSITORIES: biostudies-literature