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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.

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

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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