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CORFEDITING

Gene Editing as Therapeutic Strategy for C9ORF72 Linked ALS/FTD

Total Cost €

0

EC-Contrib. €

0

Partnership

0

Views

0

 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.

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

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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