PERSIST

Persisting Transgenesis

 Coordinatore UNIVERSITA VITA-SALUTE SAN RAFFAELE 

 Organization address address: Via Olgettina 58
city: MILANO
postcode: 20132

contact info
Titolo: Dr.
Nome: Raffaella
Cognome: Voltolini
Email: send email
Telefono: +39 02 26434765
Fax: +39 02 26434861

 Nazionalità Coordinatore Italy [IT]
 Totale costo 14˙731˙788 €
 EC contributo 11˙181˙411 €
 Programma FP7-HEALTH
Specific Programme "Cooperation": Health
 Code Call FP7-HEALTH-2007-B
 Funding Scheme CP-IP
 Anno di inizio 2009
 Periodo (anno-mese-giorno) 2009-01-01   -   2013-06-30

 Partecipanti

# participant  country  role  EC contrib. [€] 
1    UNIVERSITA VITA-SALUTE SAN RAFFAELE

 Organization address address: Via Olgettina 58
city: MILANO
postcode: 20132

contact info
Titolo: Dr.
Nome: Raffaella
Cognome: Voltolini
Email: send email
Telefono: +39 02 26434765
Fax: +39 02 26434861

IT (MILANO) coordinator 974˙143.80
2    ERASMUS UNIVERSITAIR MEDISCH CENTRUM ROTTERDAM

 Organization address address: 's Gravendijkwal 230
city: ROTTERDAM
postcode: 3015CE

contact info
Titolo: Mr.
Nome: Henk
Cognome: Isselman
Email: send email
Telefono: +31 10 7044729
Fax: +31 10 7044745

NL (ROTTERDAM) participant 935˙150.00
3    MEDIZINISCHE HOCHSCHULE HANNOVER

 Organization address address: Carl-Neuberg-Strasse 1
city: HANNOVER
postcode: 30625

contact info
Titolo: Mr.
Nome: Tobias
Cognome: Quick
Email: send email
Telefono: +49 511 532 5374
Fax: +49 511 532 4253

DE (HANNOVER) participant 826˙774.44
4    UNIVERSITY COLLEGE LONDON

 Organization address address: GOWER STREET
city: LONDON
postcode: WC1E 6BT

contact info
Titolo: Mr.
Nome: Kent
Cognome: Lee
Email: send email
Telefono: +44 20 76796296
Fax: +44 20 76796502

UK (LONDON) participant 683˙600.00
5    DEUTSCHES KREBSFORSCHUNGSZENTRUM

 Organization address address: Im Neuenheimer Feld 280
city: HEIDELBERG
postcode: 69120

contact info
Titolo: Ms.
Nome: Bettina
Cognome: Crispin
Email: send email
Telefono: +49 6221 422700
Fax: +49 6221 422708

DE (HEIDELBERG) participant 627˙468.00
6    INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM)

 Organization address address: 101 Rue de Tolbiac
city: PARIS
postcode: 75654

contact info
Titolo: Ms.
Nome: Hélène
Cognome: Brun
Email: send email
Telefono: +33 4 72138839
Fax: +33 4 472138801

FR (PARIS) participant 577˙960.00
7    VRIJE UNIVERSITEIT BRUSSEL

 Organization address address: PLEINLAAN 2
city: BRUSSEL
postcode: 1050

contact info
Titolo: Ms.
Nome: Maria
Cognome: Vereeken
Email: send email
Telefono: +32 2 6293808
Fax: +32 2 6293640

BE (BRUSSEL) participant 564˙635.40
8    GATC BIOTECH AG

 Organization address address: JAKOB STADLER PLATZ 7
city: KONSTANZ
postcode: 78467

contact info
Titolo: Mr.
Nome: Peter
Cognome: Pohl
Email: send email
Telefono: +49 7531 816047
Fax: +49 7531 8160947

DE (KONSTANZ) participant 497˙550.00
9    ASSOCIATION GENETHON

 Organization address address: RUE DE L INTERNATIONALE 1 BIS
city: EVRY
postcode: 91002

contact info
Titolo: Mr.
Nome: Jean-Philippe
Cognome: Marin
Email: send email
Telefono: +33 1 69 47 25 72
Fax: +33 1 60 77 86 98

FR (EVRY) participant 497˙200.00
10    BUNDESINSTITUT FUR IMPFSTOFFE UND BIOMEDIZINISCHE ARZNEIMITTEL

 Organization address address: PAUL-EHRLICH-STRASSE 51-59
city: LANGEN
postcode: 63225

contact info
Titolo: Ms.
Nome: Stefanie
Cognome: Larisch
Email: send email
Telefono: +49 6103 771204
Fax: +49 6103 771278

DE (LANGEN) participant 457˙905.00
11    LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN

 Organization address address: GESCHWISTER SCHOLL PLATZ 1
city: MUENCHEN
postcode: 80539

contact info
Titolo: Dr.
Nome: Anja
Cognome: Ehrhardt
Email: send email
Telefono: +49 89 5160 5270
Fax: +49 89 5160 5292

DE (MUENCHEN) participant 412˙440.00
12    UNIVERSITA DEGLI STUDI DI MODENA E REGGIO EMILIA

 Organization address address: VIA UNIVERSITA 4
city: MODENA
postcode: 41100

contact info
Titolo: Prof.
Nome: Fabio
Cognome: Prati
Email: send email
Telefono: +39 059 2055450
Fax: +39 059 2055671

IT (MODENA) participant 411˙400.00
13    ACADEMISCH ZIEKENHUIS LEIDEN

 Organization address address: Albinusdreef 2
city: LEIDEN
postcode: 2333 ZA

contact info
Titolo: Ms.
Nome: Erika
Cognome: Eikens-Bedijn
Email: send email
Telefono: +31 71 5269565
Fax: +31 71 5268275

NL (LEIDEN) participant 411˙000.00
14    ECOLE POLYTECHNIQUE FEDERALE DE LAUSANNE

 Organization address address: BATIMENT CE 3316 STATION 1
city: LAUSANNE
postcode: 1015

contact info
Titolo: Ms.
Nome: Lena
Cognome: Farneman Andersson
Email: send email
Telefono: +41 21 6391761
Fax: +41 21 6939538

CH (LAUSANNE) participant 358˙950.00
15    LUNDS UNIVERSITET

 Organization address address: Paradisgatan 5c
city: LUND
postcode: 22100

contact info
Titolo: Dr.
Nome: Jens
Cognome: Forsberg
Email: send email
Telefono: +46 46 2220575
Fax: +46 46 2220578

SE (LUND) participant 352˙551.00
16    EIDGENOESSISCHE TECHNISCHE HOCHSCHULE ZURICH

 Organization address address: Raemistrasse 101
city: ZUERICH
postcode: 8092

contact info
Titolo: Prof.
Nome: Martin
Cognome: Fussenegger
Email: send email
Telefono: +41 44 6333448
Fax: +41 44 6331234

CH (ZUERICH) participant 350˙151.00
17    EUROPAEISCHES INSTITUT FUER FORSCHUNG UND ENTWICKLUNG VON TRANSPLANTATIONSSTRATEGIEN GMBH

 Organization address address: Vollmersbachstrasse 66
city: IDAR-OBERSTEIN
postcode: 55743

contact info
Titolo: Dr.
Nome: Andrea
Cognome: Schilz
Email: send email
Telefono: +49 6781 9855232
Fax: +49 6781 9855237

DE (IDAR-OBERSTEIN) participant 328˙596.00
18    MOLECULAR MEDICINE SPA

 Organization address address: VIA OLGETTINA 58
city: MILANO
postcode: 20132

contact info
Titolo: Dr.
Nome: Enrico
Cognome: Cappelli
Email: send email
Telefono: +39 2 2127721
Fax: +39 2 21277353

IT (MILANO) participant 311˙850.00
19    VIB

 Organization address address: Rijvisschestraat 120
city: ZWIJNAARDE - GENT
postcode: 9052

contact info
Titolo: Mr.
Nome: Rik
Cognome: Audenaert
Email: send email
Telefono: +32 9 2446611
Fax: +32 9 2446610

BE (ZWIJNAARDE - GENT) participant 284˙403.60
20    Fondazione Centro San Raffaele

 Organization address address: Via Olgettina 60
city: Milano
postcode: 20132

contact info
Titolo: Dr.
Nome: Maria Rosa
Cognome: Pedrazzi
Email: send email
Telefono: 390226000000
Fax: 390226000000

IT (Milano) participant 265˙859.00
21    ACIES Consulting Group SAS

 Organization address city: LYON
postcode: 69002

contact info
Titolo: Mr.
Nome: Vincent
Cognome: Grosbois
Email: send email
Telefono: +33 4 78 92 40 01
Fax: +33 4 78 92 40 01

FR (LYON) participant 260˙470.81
22    MAX-DELBRUCK-CENTRUM FUR MOLEKULARE MEDIZIN IN DER HELMHOLTZ-GEMEINSCHAFT

 Organization address address: ROBERT ROSSLE STRASSE 10
city: BERLIN
postcode: 13125

contact info
Titolo: Mr.
Nome: Wolfgang
Cognome: Kuehlewind
Email: send email
Telefono: +49 30 94062105
Fax: +49 30 94062581

DE (BERLIN) participant 244˙503.00
23    UNIVERSITAETSKLINIKUM FREIBURG

 Organization address address: HUGSTETTER STRASSE 49
city: FREIBURG
postcode: 79106

contact info
Titolo: Mr.
Nome: Juergen
Cognome: Dreyer
Email: send email
Telefono: +49 761 270 20810
Fax: +49 761 270 18890

DE (FREIBURG) participant 207˙212.60
24    ROYAL HOLLOWAY AND BEDFORD NEW COLLEGE

 Organization address address: EGHAM HILL UNIVERSITY OF LONDON
city: EGHAM
postcode: TW20 0EX

contact info
Titolo: Ms.
Nome: Jenny
Cognome: Febry
Email: send email
Telefono: +44 1784 443019
Fax: +44 1784 475309

UK (EGHAM) participant 173˙334.00
25    CENTRO DE INVESTIGACIONES ENERGETICAS, MEDIOAMBIENTALES Y TECNOLOGICAS-CIEMAT

 Organization address address: Avenida Complutense 22
city: MADRID
postcode: 28040

contact info
Titolo: Ms.
Nome: Ana
Cognome: Collados Martin Posadillos
Email: send email
Telefono: +34 913 466 096
Fax: +34 913 466 480

ES (MADRID) participant 66˙276.00
26    CHEMOTHERAPEUTISCHES FORSCHUNGSINSTITUT GEORG-SPEYER-HAUS STIFTUNG

 Organization address address: PAUL EHRLICH STRASSE 42-44
city: FRANKFURT
postcode: 60596

contact info
Titolo: Mr.
Nome: Robert
Cognome: Dornberger
Email: send email
Telefono: +49 69 6339 5333
Fax: +49 69 6339 5297

DE (FRANKFURT) participant 60˙000.00
27    CHARITE - UNIVERSITAETSMEDIZIN BERLIN

 Organization address address: Chariteplatz 1
city: BERLIN
postcode: 10117

contact info
Titolo: Mr.
Nome: Eveline
Cognome: Frädorf
Email: send email
Telefono: -450576005
Fax: -450576935

DE (BERLIN) participant 20˙498.16
28    ACIES SAS

 Organization address address: RUE DE LA REPUBLIQUE 69
city: LYON
postcode: 69002

contact info
Titolo: Mr.
Nome: Patrick
Cognome: Duvarry
Email: send email
Telefono: -72983971
Fax: -78923972

FR (LYON) participant 19˙529.19
29    NOVAMEN SAS

 Organization address city: LYON
postcode: 69002

contact info
Titolo: Mr.
Nome: Vincent
Cognome: Grosbois
Email: send email
Telefono: +33 4 78 92 40 01
Fax: +33 4 78 92 40 01

FR (LYON) participant 0.00

Mappa


 Word cloud

Esplora la "nuvola delle parole (Word Cloud) per avere un'idea di massima del progetto.

innovative    persisting    expression    genome    therapeutic    haemophilias    scientists    transgene    immune    advantage    trials    disease    engineering    integration    vector    viral    storage    site    disorders    clinical    immunodeficiencies    transfer    dna    efficiency    hscs    medicines    gene    persist    therapy    innovations    vectors    cells    inherited    cancer    genetic    deadly    line    types    cell    discovery    technologies    lentiviral    diseases   

 Obiettivo del progetto (Objective)

'For many disabling or fatal diseases, there is pre-clinical or clinical evidence of the potential therapeutic efficacy of gene therapy and, yet, the limitations of current gene transfer technologies have prevented success or even caused serious adverse events leading to termination of trials. PERSIST will explore the use of highly innovative gene-modifying and delivery technologies and capitalize on recent discoveries in gene expression control to develop radical solutions to the problem of precisely controlling the fate and expression of exogenous genetic information in gene therapy with applications in these and other deadly diseases. Our proposal combines 20 of Europe’s outstanding experts from 8 countries in the field of genetic engineering for persisting gene expression. Partners have pioneered the use of Zinc Finger Nucleases, engineered recombinases and transposases for gene targeting, synthetic promoters, epigenetic switches and micro-RNA for regulating gene expression, developed state-of-the-art gene delivery platforms based on lentiviral, AAV and gutless adenoviral vectors, conducted front-line clinical trials, and productively collaborated in previous FP projects. PERSIST includes 16 work packages of which 6 focus on vector innovations (part A: emerging technologies), 6 on applications & evaluation (part B), 1 on process development and the remaining 2 on training/dissemination and project management including IPR issues. Targeting deadly diseases, such as inherited immunodeficiencies, storage disorders and haemophilias, PERSIST is in line with the Strategic Research Agenda (SRA) of the Innovative Medicines Initiative (IMI) and will result in: faster discovery and development of better medicines; more attractive professional environment for scientists; better European expertise and know-how to attract biomedical R&D investment in Europe; and a stronger competitive advantage for SMEs, spin-offs and start-ups to enhance Europe’s economy.'

Descrizione progetto (Article)

Replacement of a faulty mutated gene with a functional therapeutic piece of DNA requires a carrier or vector. Insertion of the transgene with accuracy in terms of cell and site in the genome is crucial. There must also be tight control of transgene expression to avoid unwanted genetic side-effects.

The EU-funded 'Persisting transgenesis' (http://www.persist-project.eu/ (PERSIST)) project has researched every angle to advance genetic engineering and persisting gene expression. Severe diseases tackled included inherited immunodeficiencies, storage disorders, haemophilias, as well as some types of cancer.

Innovations included surface engineering of lentiviral vectors. This approach produced new types of vehicle for targeted gene transfer into human blood cell producing (haematopoietic) stem cells (HSCs). This versatile tool can target gene transfer to immune and endothelial cells for treating cancer and immune system disorders.

A non-viral alternative explored by the PERSIST team is the Sleeping Beauty transposon, a DNA sequence that can change its position in the recipient's genome. Careful choice of target site promises to negate the inherent disadvantage of the technique due to its random action.

The scientists have set up integration site landscapes for various viral vectors. Prospective monitoring of vector integration sites in clinical gene therapy studies is feasible for detecting possible side effects of gene therapy in real-time.

PERSIST developed site-specific gene correction using HSCs with an efficiency sufficient for clinical application. This is the first technology of its kind and with refinement the partners anticipate its incorporation into clinical studies in five years. Indicative of the proximity to clinical practice, one of the Chronic Granulomatous Disease vectors has entered Phase I/II trials.

Optimisation of the methods developed will increase the efficiency of lentiviral vector manufacture. This has the added advantage of reducing the price for the patient as well as the high cost of running clinical trials.

Diseases targeted by PERSIST research include those with a high social burden and is edging into the massive infectious disease arena. The outcome will be more rapid discovery and development of better therapies to meet Europe's health challenges.

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