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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.

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

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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