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UPGRADE SIGNED

Unlocking Precision Gene Therapy

Total Cost €

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EC-Contrib. €

0

Partnership

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 UPGRADE project word cloud

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

definitive    off    conventional    cell    stringently    opening    editing    models    radical    wasting    strategies    vectors    precise    combine    disruptive    mutagenesis    cellular    risk    safety    heart    profile    full    evasion    unmet    amp    activate    treatment    correction    realize    transfer    vehicles    specificity    cure    hurdles    recode    reached    treatments    poor    groups    disease    scope    insertion    basis    sequence    shown    otherwise    generate    silence    medical    tissues    muscle    portable    efficacy    pathogenesis    disorders    transgene    clinical    skeletal    amps    gene    versatile    viral    incurable    liver    prototypes    durability    indications    validated    entering    limited    site    storage    upgrade    patient    diseases    tools    paradigmatic    blood    hematopoiesis    tissue    efficiency    epigenome    insertional    limitations    remarkable    tackling    retina    hypercholesterolemia    broaden    medicinal    immune    seven    components    complementing    precision    overcome    genome    market    therapy    technologies    solutions   

Project "UPGRADE" data sheet

The following table provides information about the project.

Coordinator
FONDAZIONE TELETHON 

Organization address
address: VIA VARESE 16/B
city: ROMA
postcode: 185
website: www.telethon.it

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 Italy [IT]
 Total cost 14˙996˙955 €
 EC max contribution 14˙995˙830 € (100%)
 Programme 1. H2020-EU.3.1.3. (Treating and managing disease)
 Code Call H2020-SC1-2018-Single-Stage-RTD
 Funding Scheme RIA
 Starting year 2019
 Duration (year-month-day) from 2019-01-01   to  2023-12-31

 Partnership

Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 
1    FONDAZIONE TELETHON IT (ROMA) coordinator 5˙027˙968.00
2    UNIVERSIDAD POMPEU FABRA ES (BARCELONA) participant 2˙767˙377.00
3    UNIVERSITA DEGLI STUDI DI TRENTO IT (TRENTO) participant 1˙333˙798.00
4    VRIJE UNIVERSITEIT BRUSSEL BE (BRUSSEL) participant 1˙328˙412.00
5    THE GENERAL HOSPITAL CORPORATION US (BOSTON MA) participant 1˙217˙710.00
6    UNIVERSITE DE NANTES FR (NANTES CEDEX 1) participant 1˙107˙500.00
7    TECHNISCHE UNIVERSITAET DRESDEN DE (DRESDEN) participant 1˙107˙000.00
8    LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN DE (MUENCHEN) participant 1˙106˙062.00

Map

 Project objective

Gene therapy has recently shown remarkable potential to offer definitive treatments for otherwise incurable diseases. Currently, seven gene therapy products have reached the market and many more are entering clinical testing for selected indications. Moreover, emerging technologies for targeted gene editing are complementing the scope of conventional gene transfer, opening the way to precise gene correction and making possible to silence, activate or recode any sequence of interest in the genome. However, in order to realize the full potential of these strategies and broaden application of gene therapy, the field has to solve several major hurdles, including: risk of insertional mutagenesis by gene transfer vectors, limited efficiency and durability of some gene correction strategies, off target effects of editing tools, poor tissue targeting and immune response to editing components and delivery vehicles. UPGRADE will offer radical new solutions to overcome these hurdles. We will exploit and further develop disruptive new technologies for precision gene and epigenome editing and for site-specific transgene insertion, and stringently characterize their specificity and cellular responses. We will combine these improved technologies with advanced viral and non-viral vectors enabling cell/tissue targeting and immune evasion, to generate prototypes of advanced medicinal products (AMP). The safety and efficacy profile of each AMP will be stringently validated in tissues (hematopoiesis, heart and skeletal muscle, liver, retina) and disease models (muscle wasting, storage and blood disorders, hypercholesterolemia) paradigmatic for unmet medical need and potential long-term cure, if the limitations of current gene therapy strategies are overcome. These AMPs represent versatile products portable to the treatment of several other diseases because of related pathogenesis or correction strategies, thus providing the basis for tackling diseases affecting large patient groups.

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

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