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Investigation of original multivalent iminosugars as pharmacological chaperones for the treatment of Gaucher disease.
- Source :
-
Carbohydrate research [Carbohydr Res] 2016 Jun 24; Vol. 429, pp. 98-104. Date of Electronic Publication: 2016 Mar 16. - Publication Year :
- 2016
-
Abstract
- Multivalent iminosugars conjugated with a morpholine moiety and/or designed as prodrugs have been prepared and evaluated as new classes of pharmacological chaperones for the treatment of Gaucher disease. This study further confirms the interest of the prodrug concept and shows that the addition of a lysosome-targeting morpholine unit into iminosugar cluster structures has no significant impact on the chaperone activity on Gaucher cells.<br /> (Copyright © 2016 Elsevier Ltd. All rights reserved.)
- Subjects :
- Click Chemistry
Enzyme Activation drug effects
Enzyme Reactivators pharmacology
Fibroblasts enzymology
Fibroblasts pathology
Gaucher Disease drug therapy
Gaucher Disease enzymology
Gaucher Disease pathology
Glucosylceramidase deficiency
Humans
Imino Sugars pharmacology
Kinetics
Lysosomes enzymology
Molecular Targeted Therapy
Morpholines chemistry
Prodrugs pharmacology
Protein Binding
Protein Folding
Enzyme Reactivators chemical synthesis
Fibroblasts drug effects
Glucosylceramidase chemistry
Imino Sugars chemical synthesis
Lysosomes drug effects
Prodrugs chemical synthesis
Subjects
Details
- Language :
- English
- ISSN :
- 1873-426X
- Volume :
- 429
- Database :
- MEDLINE
- Journal :
- Carbohydrate research
- Publication Type :
- Academic Journal
- Accession number :
- 27063390
- Full Text :
- https://doi.org/10.1016/j.carres.2016.03.007