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Gene Editing as Therapeutic Strategy for C9ORF72 Linked ALS/FTD

Total Cost €


EC-Contrib. €






 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.

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

Project "CORFEDITING" data sheet

The following table provides information about the project.


Organization address
postcode: S10 2TN

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


Take a look of project's partnership.

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


 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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