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How do Pharmacological Chaperones work? Molecular basis of the actions of glycomimetics on keyglycosidases involved in lysosomal storage disorders

Total Cost €


EC-Contrib. €






 GlycoPCs project word cloud

Explore the words cloud of the GlycoPCs project. It provides you a very rough idea of what is the project "GlycoPCs" about.

pcs    principles    bind    deepen    mechanisms    poorly    chaperones    powerful    mass    until    cellular    casal    drugs    diseases    nmr    lysosome    resolution    mechanism    lsds    therapeutic    besides    structural    molecules    resonance    discovered    spectrometry    small    glycosidases    pc    library    pharmacological    lack    simulations    wild    underpinning    dysfunction    skills    folding    enzymes    disorders    enzyme    destabilise    gaucher    prevalent    stabilize    nuclear    ligands    rational    mutations    transportation    ms    glycosidase    symptoms    employs    replace    group    severe    researcher    dynamics    leads    rare    operated    effectiveness    metabolic    magnetic    accumulation    mutant    dr    translocation    storage    inherited    molecular    guiding    fabry    lysosomes    protocol    reducing    substrate    discovery    stabilization    precluding    combined    understand    lysosomal    methodological    proper    efficient    criteria    originate    combining    market    3d    action    drug    therapies    adding    treatment    trained   

Project "GlycoPCs" data sheet

The following table provides information about the project.


Organization address
postcode: NR4 7TJ

contact info
title: n.a.
name: n.a.
surname: n.a.
function: n.a.
email: n.a.
telephone: n.a.
fax: n.a.

 Coordinator Country United Kingdom [UK]
 Total cost 183˙454 €
 EC max contribution 183˙454 € (100%)
 Programme 1. H2020-EU.1.3.2. (Nurturing excellence by means of cross-border and cross-sector mobility)
 Code Call H2020-MSCA-IF-2017
 Funding Scheme MSCA-IF-EF-ST
 Starting year 2018
 Duration (year-month-day) from 2018-10-01   to  2020-09-30


Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 
1    UNIVERSITY OF EAST ANGLIA UK (NORWICH) coordinator 183˙454.00


 Project objective

Lysosomal storage disorders (LSDs) are a group of 50 rare inherited metabolic disorders that in many cases originate from mutations that destabilise glycosidase 3D folding precluding its transportation to the lysosomes, which leads to substrate accumulation at the lysosomes and cellular dysfunction, with severe symptoms. An emerging therapeutic approach employs small molecules, called pharmacological chaperones (PCs). PCs bind and stabilize the folding of mutant lysosomal enzymes, allowing proper cellular translocation to the lysosome, reducing substrate accumulation. However, there is still no drug already on the market based on this concept and the PCs discovered until now lack the necessary effectiveness to replace other therapies. One important reason is that the complex mechanisms underpinning the enzyme stabilization operated by chaperones are poorly understood in structural terms and currently under debate. Understanding the mechanism will provide more rational criteria and guiding principles for the design of improved PC drugs. In this proposal we are interested in providing novel structural approaches to understand the mechanism of action of new PCs for efficient treatment in Gaucher and Fabry diseases, as two of the more prevalent LSDs. We will develop a powerful high-resolution combined protocol including mass spectrometry (MS) and nuclear magnetic resonance (NMR) to: (i) provide a novel methodological approach for the discovery of PCs, (ii) apply the novel protocol to a small library of promising new ligands, and (iii) deepen our understanding of the mechanism of action of PCs in structural terms by combining the novel MS/NMR protocol with very long molecular dynamics simulations of wild-type and mutant glycosidases. Besides the potential for high-impact of the project, it will also allow the experienced researcher, Dr E.Casal, to be trained in a wide range of new skills, adding to her strong previous experience in MS.

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The information about "GLYCOPCS" are provided by the European Opendata Portal: CORDIS opendata.

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