GCase Enhancers: A Potential Therapeutic Option for Gaucher Disease and Other Neurological Disorders.
Gaucher disease
Parkinson disease
allosteric chaperones
iminosugars
lysosomal storage disorders
pharmacological chaperones
Journal
Pharmaceuticals (Basel, Switzerland)
ISSN: 1424-8247
Titre abrégé: Pharmaceuticals (Basel)
Pays: Switzerland
ID NLM: 101238453
Informations de publication
Date de publication:
02 Jul 2022
02 Jul 2022
Historique:
received:
10
06
2022
revised:
28
06
2022
accepted:
29
06
2022
entrez:
27
7
2022
pubmed:
28
7
2022
medline:
28
7
2022
Statut:
epublish
Résumé
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 the scientific community in the last 7 years (since 2015) in order to identify new PCs for the GCase enzyme, which have been mainly identified among glycomimetic-based compounds.
Identifiants
pubmed: 35890122
pii: ph15070823
doi: 10.3390/ph15070823
pmc: PMC9325019
pii:
doi:
Types de publication
Journal Article
Review
Langues
eng
Subventions
Organisme : Università di Firenze and Fondazione CR Firenze ("Bando congiunto per il finanziamento di pro-getti competitivi sulle malattie neurodegenerative 2018")
ID : Acronym: MuTaParGa
Organisme : Regione Toscana (Bando Salute 2018)
ID : Acronym: LysoLate
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