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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.

efficiency patient therapy amps technologies components insertional heart scope immune genome site treatment activate unmet combine disruptive reached limited clinical amp strategies versatile definitive remarkable limitations insertion realize off skeletal profile hypercholesterolemia prototypes overcome transgene portable otherwise cell poor medicinal complementing tissues recode indications treatments seven specificity risk transfer entering hematopoiesis radical tackling conventional diseases safety tools tissue editing efficacy paradigmatic validated epigenome precision medical hurdles viral cellular upgrade broaden liver vectors basis gene solutions market vehicles disorders cure pathogenesis durability mutagenesis evasion muscle silence full stringently correction disease groups opening models generate wasting storage precise retina incurable sequence shown 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 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.	IT (ROMA)	coordinator	5˙027˙968.00
2	UNIVERSIDAD POMPEU FABRA UNIVERSIDAD POMPEU FABRA Organization address address: PLACA DE LA MERCE, 10-12 city: BARCELONA postcode: 8002 website: www.upf.edu contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	ES (BARCELONA)	participant	2˙767˙377.00
3	UNIVERSITA DEGLI STUDI DI TRENTO UNIVERSITA DEGLI STUDI DI TRENTO Organization address address: VIA CALEPINA 14 city: TRENTO postcode: 38122 website: www.unitn.it contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	IT (TRENTO)	participant	1˙333˙798.00
4	VRIJE UNIVERSITEIT BRUSSEL VRIJE UNIVERSITEIT BRUSSEL Organization address address: PLEINLAAN 2 city: BRUSSEL postcode: 1050 website: www.vub.ac.be contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	BE (BRUSSEL)	participant	1˙328˙412.00
5	THE GENERAL HOSPITAL CORPORATION THE GENERAL HOSPITAL CORPORATION Organization address address: FRUIT STREET 55 city: BOSTON MA postcode: 2114 website: n.a. contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	US (BOSTON MA)	participant	1˙217˙710.00
6	UNIVERSITE DE NANTES UNIVERSITE DE NANTES Organization address address: QUAI DE TOURVILLE 1 city: NANTES CEDEX 1 postcode: 44035 website: www.univ-nantes.fr contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	FR (NANTES CEDEX 1)	participant	1˙107˙500.00
7	TECHNISCHE UNIVERSITAET DRESDEN TECHNISCHE UNIVERSITAET DRESDEN Organization address address: HELMHOLTZSTRASSE 10 city: DRESDEN postcode: 1069 website: http://www.tu-dresden.de/ contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	DE (DRESDEN)	participant	1˙107˙000.00
8	LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN Organization address address: GESCHWISTER SCHOLL PLATZ 1 city: MUENCHEN postcode: 80539 website: www.uni-muenchen.de contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	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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