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CORFEDITING

Gene Editing as Therapeutic Strategy for C9ORF72 Linked ALS/FTD

Total Cost €

0

EC-Contrib. €

0

Partnership

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 CORFEDITING project word cloud

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

care    deficits    gene    motor    proof    parkinson    huntington    72    economic    efforts    designing    majority    cas9    fatal    mouse    utmost    vectors    treatment    individuals    prospect    als    successful    options    genetic    ftd    cns    expansion    hexanucleotide    continues    models    amenable    patients    remove    anticipate    mns    chromosome    region    therapeutic    discoveries    neuromuscular    disorders    lateral    goes    dementia    alzheimer    potentiates    death    neurological    translated    afflicted    respiratory    benefit    neurons    pathogenic    disease    multiple    optimise    spectrum    g4c2    scenarios    devastating    erc    burden    beneficial    frontotemporal    expansions    frame    modification    degeneration    therapy    cerebellar    neurodegenerative    made    repeat    reading    strategy    worldwide    significantly    amyotrophic    expensive    treat    coding    progressive    upper    diseases    sclerosis    lower    crispr    ataxias    ultimate    c9orf72    suggests    selectively    containing   

Project "CORFEDITING" data sheet

The following table provides information about the project.

Coordinator
THE UNIVERSITY OF SHEFFIELD 

Organization address
address: FIRTH COURT WESTERN BANK
city: SHEFFIELD
postcode: S10 2TN
website: www.shef.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 149˙995 €
 EC max contribution 149˙995 € (100%)
 Programme 1. H2020-EU.1.1. (EXCELLENT SCIENCE - European Research Council (ERC))
 Code Call ERC-2016-PoC
 Funding Scheme ERC-POC
 Starting year 2017
 Duration (year-month-day) from 2017-06-01   to  2018-11-30

 Partnership

Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 
1    THE UNIVERSITY OF SHEFFIELD UK (SHEFFIELD) coordinator 149˙995.00

Map

 Project objective

Expansion of a hexanucleotide repeat G4C2 in the non-coding region of chromosome 9 open reading frame 72 (C9orf72) is the most common genetic cause for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). ALS is a fatal condition characterized by progressive motor deficits, degeneration of upper and lower motor neurons (MNs) and death from neuromuscular respiratory failure in the majority of afflicted individuals within 3-5 years. Currently, the economic burden of care and treatment for patients with ALS/FTD is expensive and continues to significantly rise in Europe and worldwide. While significant genetic discoveries have been made in the field, they have not yet translated to treatment options for patients with ALS and FTD. Thus, research efforts aimed at identifying therapeutic targets are of the utmost importance to enable therapeutic development for these devastating disorders. In this ERC Proof of Concept project, we will design, optimise and test gene therapy vectors containing CRISPR/Cas9 system to selectively remove the pathogenic ALS/FTD-related C9orf72 hexanucleotide repeat expansion in mouse models of C9orf72-related ALS, with the ultimate aim of designing a therapy for patients with C9orf72-related ALS/FTD. The ultimate benefit of this approach goes far beyond just ALS/FTD however. A successful CNS gene therapy for C9orf72 related disease potentiates the prospect of developing similar approaches to treat multiple disease scenarios amenable to gene modification. Indeed, growing evidence suggests that C9orf72 repeat expansions also contribute to a wide spectrum of neurodegenerative diseases such as Alzheimer’s, Huntington’s, multiple sclerosis, Parkinson’s disease and cerebellar ataxias. We therefore anticipate that our strategy could be beneficial for other neurological conditions.

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

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