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Opendata, web and dolomites

UPGRADE SIGNED

Unlocking Precision Gene Therapy

Total Cost €

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EC-Contrib. €

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Partnership

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Views

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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.

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

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

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