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UPGRADE SIGNED

Unlocking Precision Gene Therapy

Total Cost €

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EC-Contrib. €

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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.

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

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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