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GlycoPCs SIGNED

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

Total Cost €

0

EC-Contrib. €

0

Partnership

0

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 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.

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

Project "GlycoPCs" data sheet

The following table provides information about the project.

Coordinator
UNIVERSITY OF EAST ANGLIA 

Organization address
address: EARLHAM ROAD
city: NORWICH
postcode: NR4 7TJ
website: http://www.uea.ac.uk

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

 Partnership

Take a look of project's partnership.

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

Map

 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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