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LYSOBONE SIGNED

Cellular and molecular analysis of the skeletal pathologies associated with mucopolysaccharidosis-VI (MPS-VI)

Total Cost €

0

EC-Contrib. €

0

Partnership

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 LYSOBONE project word cloud

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

storage    scientific    clinically    mutations    audiences    intellectual    press    deficiency    union    relevance    ert    meetings    preliminary    genetics    skeletal    unraveled    final    osteoporosis    types    mechanisms    releases    mucopolysaccharidosis    expert    skills    encoding    intensive    data    pathogenic    disseminate    provides    lysosomal    disorder    researcher    transfer    mps    administrative    transferred    vi    acquired    therapy    experimental    disorders    pathologies    glycosaminoglycan    patient    mice    arsb    phenotype    unravel    cellular    broad    question    previously    degrading    publications    mouse    handling    influence    excellent    specialized    postdoctoral    severity    financial    replacement    closely    writing    predominantly    host    understand    training    defects    model    generate    prevalent    regarding    personal    molecular    me    osteoarthritis    tool    methodological    organization    caused    arylsulfatase    deficient    enzyme    goals    cell    gene    linked    primary    remodelling    propagate    conferences   

Project "LYSOBONE" data sheet

The following table provides information about the project.

Coordinator		 UNIVERSITAETSKLINIKUM HAMBURG-EPPENDORF 

Organization address
address: Martinistrasse 52
city: HAMBURG
postcode: 20251
website: www.uke.uni-hamburg.de

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 Germany [DE]
 Total cost 159˙460 €
 EC max contribution 159˙460 € (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 2018-03-01   to  2020-02-29

 Partnership

Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 
1    UNIVERSITAETSKLINIKUM HAMBURG-EPPENDORF DE (HAMBURG) coordinator 159˙460.00

Map

 Project objective

Mucopolysaccharidosis-VI (MPS-VI) is a lysosomal storage disorder predominantly affecting skeletal remodelling and caused by pathogenic mutations in the ARSB gene, encoding the glycosaminoglycan-degrading enzyme arylsulfatase B. Preliminary analysis of Arsb-deficient mice unraveled a skeletal phenotype of similar severity and with lysosomal storage defects in different skeletal cell types. This Arsb-deficient mouse model therefore provides an excellent tool to understand the skeletal and non-skeletal pathologies related to MPS-VI and to address the clinically relevant question, if enzyme replacement therapy (ERT) can influence the existing (non-)skeletal pathologies in lysosomal storage disorders. Moreover, another major objective is to unravel the cellular consequences of Arsb-deficiency on a molecular level and to investigate mechanisms of ARSB uptake and lysosomal delivery in different primary cell types of Arsb-deficient mice. This project will therefore certainly generate knowledge with relevance for other (skeletal) disorders as well, as the cellular defects in MPS-VI are closely linked to that of two of the most prevalent disorders in the European Union, i.e. osteoporosis and osteoarthritis. Next to its scientific goals, this proposal aims to offer a broad personal training program for me as a postdoctoral researcher as well. Handling this project will not only offer an intensive scientific training (intellectual-methodological), but also improve my skills regarding project management (administrative-financial) and writing. As a two-way transfer, my previously acquired knowledge on the genetics of skeletal disorders and related experimental skills will be transferred to the host organization. A final goal of this proposal is to disseminate the acquired data towards specialized scientific audiences (scientific conferences and publications) and propagate these data towards non-expert audiences (patient meetings, press releases) as well.

 Publications

year authors and title journal last update
List of publications.
2020 Gretl Hendrickx, Tatyana Danyukova, Anke Baranowsky, Tim Rolvien, Alexandra Angermann, Michaela Schweizer, Johannes Keller, Jörg Schröder, Catherine Meyer-Schwesinger, Nicole Muschol, Chiara Paganini, Antonio Rossi, Michael Amling, Sandra Pohl, Thorsten Schinke
Enzyme replacement therapy in mice lacking arylsulfatase B targets bone remodeling cells, but not chondrocytes
published pages: , ISSN: 0964-6906, DOI: 10.1093/hmg/ddaa006 		Human Molecular Genetics 2020-02-13
2018 Sandra Pohl, Alexandra Angermann, Anke Jeschke, Gretl Hendrickx, Timur A Yorgan, Georgia Makrypidi-Fraune, Anita Steigert, Sonja C Kuehn, Tim Rolvien, Michaela Schweizer, Till Koehne, Mona Neven, Olga Winter, Renata Voltolini Velho, Joachim Albers, Thomas Streichert, Jan M Pestka, Christina Baldauf, Sandra Breyer, Ralf Stuecker, Nicole Muschol, Timothy M Cox, Paul Saftig, Chiara Paganini, Antonio Rossi, Michael Amling, Thomas Braulke, Thorsten Schinke
The Lysosomal Protein Arylsulfatase B Is a Key Enzyme Involved in Skeletal Turnover
published pages: , ISSN: 0884-0431, DOI: 10.1002/jbmr.3563 		Journal of Bone and Mineral Research 2019-06-11

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

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