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CORFEDITING

Gene Editing as Therapeutic Strategy for C9ORF72 Linked ALS/FTD

Total Cost €

0

EC-Contrib. €

0

Partnership

0

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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.

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

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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