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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.

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

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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