GCase Enhancers: A Potential Therapeutic Option for Gaucher Disease and Other Neurological Disorders.

ABSTRACT: Pharmaceutical chaperones (PCs) are small compounds able to bind and stabilize misfolded proteins, allowing them to recover their native folding and thus their biological activity. In particular, lysosomal storage disorders (LSDs), a class of metabolic disorders due to genetic mutations that result in misfolded lysosomal enzymes, can strongly benefit from the use of PCs able to facilitate their translocation to the lysosomes. This results in a recovery of their catalytic activity. No PC for the GCase enzyme (lysosomal acid-β-glucosidase, or glucocerebrosidase) has reached the market yet, despite the importance of this enzyme not only for Gaucher disease, the most common LSD, but also for neurological disorders, such as Parkinson's disease. This review aims to describe the efforts made by t

SUBMITTER: Martinez-Bailen M 

PROVIDER: S-EPMC9325019 | biostudies-literature | 2022 Jul

REPOSITORIES: biostudies-literature