Role of misfolding in rare enzymatic deficits and use of pharmacological chaperones as therapeutic approach

Role of misfolding in rare enzymatic deficits and use of pharmacological chaperones as therapeutic approach

Cells have evolved sophisticated molecular control systems to maximize the efficiency of the folding process. However, any subtle alteration of the environment or the protein can lead to misfolding or affect the conformational plasticity of the native states. It has been widely demonstrated that mis...

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Bibliographic Details
Main Authors: Gioena Pampalone, Silvia Grottelli, Leonardo Gatticchi, Emilia Maria Lombardi, Ilaria Bellezza, Barbara Cellini
Format: Article
Language:English
Published: IMR Press 2021-12-01
Series:Frontiers in Bioscience-Landmark
Subjects:
misfolding
parmacological chaperones
gaucher disease
phenylketonuria
primary hyperoxaluria
Online Access:https://www.imrpress.com/journal/FBL/26/12/10.52586/5056

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