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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.

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

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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