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DREAM SIGNED

Dux4-Regulated Expression and Activity by MATR3

Total Cost €

0

EC-Contrib. €

0

Partnership

0

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 DREAM project word cloud

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

laboratory    muscular    transcriptional    molecule    body    dystrophy    afflicts    activation    loci    examine    sexes    embryogenesis    elucidate    mechanism    characterizing    direct    cytotoxic    models    regulator    trials    degeneration    activates    ages    toxic    stages    identification    stem    pro    function    clinical    indiscriminately    double    continues    dux4    data    matr3    clarify    preliminary    patients    protects    wasting    cure    homeobox    interacting    tissues    toxicity    muscle    aberrant    treatment    regulates    domain    myogenic    pathogenesis    preventing    action    expression    compelling    inhibiting    despite    adults    closer    mis    tools    molecular    treat    corollary    rational    innovation    strategy    binding    genes    restricted    option    caused    therapeutic    inhibitor    hypothesize    first    cells    subjects    peptidomimetic    healthy    block    fshd    representing    blocking    facioscapulohumeral    disease    apoptotic    prevalent    prevent    silenced    children    transcription   

Project "DREAM" data sheet

The following table provides information about the project.

Coordinator
OSPEDALE SAN RAFFAELE SRL 

Organization address
address: VIA OLGETTINA 60
city: MILANO
postcode: 20132
website: www.hsr.it

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 Italy [IT]
 Total cost 183˙473 €
 EC max contribution 183˙473 € (100%)
 Programme 1. H2020-EU.1.3.2. (Nurturing excellence by means of cross-border and cross-sector mobility)
 Code Call H2020-MSCA-IF-2019
 Funding Scheme MSCA-IF-EF-SE
 Starting year 2020
 Duration (year-month-day) from 2020-05-01   to  2022-04-30

 Partnership

Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 
1    OSPEDALE SAN RAFFAELE SRL IT (MILANO) coordinator 183˙473.00

Map

 Project objective

Facioscapulohumeral muscular dystrophy (FSHD) is the most prevalent muscle disease that indiscriminately afflicts children and adults of all ages and both sexes. Despite several clinical trials, there continues to be no cure or therapeutic option available to FSHD patients. FSHD is caused by aberrant myogenic expression of the transcription factor double homeobox 4 (DUX4). In healthy subjects, DUX4 expression is restricted to stem cells and early stages of embryogenesis while being silenced in most tissues of the body. In FSHD, DUX4 mis-expression activates a pro-apoptotic transcriptional program leading to muscle wasting. Compelling data obtained in our laboratory identified MATR3 as the first direct DUX4 inhibitor and a therapeutic molecule. The identification of a novel regulator of DUX4 function now allows us to examine the potential of inhibiting the cytotoxic activity of DUX4 as a therapeutic strategy to treat FSHD, and represents the innovation of this proposal. The goal of the proposal is to fully elucidate the molecular mechanism through which MATR3 regulates DUX4 function in order to develop novel therapeutic approaches for blocking the aberrant activity of DUX4 in FSHD. Based on our preliminary data, I hypothesize that MATR3 protects from DUX4-induced toxicity by inhibiting DUX4 binding to target loci with the end result of preventing transcriptional activation of genes toxic to muscle cells. Characterizing MATR3 mechanism of action will provide possible therapeutic tools that we will evaluate in relevant models of FSHD. As a corollary, we propose that a peptidomimetic molecule representing the DUX4 interacting domain of MATR3 could be used to prevent the toxic effects of DUX4 expression in muscle cells from FSHD patients. This work is significant as it will clarify the molecular pathogenesis of the disease and bring us closer to a rational treatment to block muscle degeneration in FSHD.

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

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