PERSIST
Persisting Transgenesis
| Coordinatore | UNIVERSITA VITA-SALUTE SAN RAFFAELE
Organization address
address: Via Olgettina 58 contact info |
| 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
| # | ||||
|---|---|---|---|---|
| 1 |
UNIVERSITA VITA-SALUTE SAN RAFFAELE
Organization address
address: Via Olgettina 58 contact info |
IT (MILANO) | coordinator | 974˙143.80 |
| 2 |
ERASMUS UNIVERSITAIR MEDISCH CENTRUM ROTTERDAM
Organization address
address: 's Gravendijkwal 230 contact info |
NL (ROTTERDAM) | participant | 935˙150.00 |
| 3 |
MEDIZINISCHE HOCHSCHULE HANNOVER
Organization address
address: Carl-Neuberg-Strasse 1 contact info |
DE (HANNOVER) | participant | 826˙774.44 |
| 4 |
UNIVERSITY COLLEGE LONDON
Organization address
address: GOWER STREET contact info |
UK (LONDON) | participant | 683˙600.00 |
| 5 |
DEUTSCHES KREBSFORSCHUNGSZENTRUM
Organization address
address: Im Neuenheimer Feld 280 contact info |
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 contact info |
FR (PARIS) | participant | 577˙960.00 |
| 7 |
VRIJE UNIVERSITEIT BRUSSEL
Organization address
address: PLEINLAAN 2 contact info |
BE (BRUSSEL) | participant | 564˙635.40 |
| 8 |
GATC BIOTECH AG
Organization address
address: JAKOB STADLER PLATZ 7 contact info |
DE (KONSTANZ) | participant | 497˙550.00 |
| 9 |
ASSOCIATION GENETHON
Organization address
address: RUE DE L INTERNATIONALE 1 BIS contact info |
FR (EVRY) | participant | 497˙200.00 |
| 10 |
BUNDESINSTITUT FUR IMPFSTOFFE UND BIOMEDIZINISCHE ARZNEIMITTEL
Organization address
address: PAUL-EHRLICH-STRASSE 51-59 contact info |
DE (LANGEN) | participant | 457˙905.00 |
| 11 |
LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN
Organization address
address: GESCHWISTER SCHOLL PLATZ 1 contact info |
DE (MUENCHEN) | participant | 412˙440.00 |
| 12 |
UNIVERSITA DEGLI STUDI DI MODENA E REGGIO EMILIA
Organization address
address: VIA UNIVERSITA 4 contact info |
IT (MODENA) | participant | 411˙400.00 |
| 13 |
ACADEMISCH ZIEKENHUIS LEIDEN
Organization address
address: Albinusdreef 2 contact info |
NL (LEIDEN) | participant | 411˙000.00 |
| 14 |
ECOLE POLYTECHNIQUE FEDERALE DE LAUSANNE
Organization address
address: BATIMENT CE 3316 STATION 1 contact info |
CH (LAUSANNE) | participant | 358˙950.00 |
| 15 |
LUNDS UNIVERSITET
Organization address
address: Paradisgatan 5c contact info |
SE (LUND) | participant | 352˙551.00 |
| 16 |
EIDGENOESSISCHE TECHNISCHE HOCHSCHULE ZURICH
Organization address
address: Raemistrasse 101 contact info |
CH (ZUERICH) | participant | 350˙151.00 |
| 17 |
EUROPAEISCHES INSTITUT FUER FORSCHUNG UND ENTWICKLUNG VON TRANSPLANTATIONSSTRATEGIEN GMBH
Organization address
address: Vollmersbachstrasse 66 contact info |
DE (IDAR-OBERSTEIN) | participant | 328˙596.00 |
| 18 |
MOLECULAR MEDICINE SPA
Organization address
address: VIA OLGETTINA 58 contact info |
IT (MILANO) | participant | 311˙850.00 |
| 19 |
VIB
Organization address
address: Rijvisschestraat 120 contact info |
BE (ZWIJNAARDE - GENT) | participant | 284˙403.60 |
| 20 |
Fondazione Centro San Raffaele
Organization address
address: Via Olgettina 60 contact info |
IT (Milano) | participant | 265˙859.00 |
| 21 |
ACIES Consulting Group SAS
Organization address
city: LYON contact info |
FR (LYON) | participant | 260˙470.81 |
| 22 |
MAX-DELBRUCK-CENTRUM FUR MOLEKULARE MEDIZIN IN DER HELMHOLTZ-GEMEINSCHAFT
Organization address
address: ROBERT ROSSLE STRASSE 10 contact info |
DE (BERLIN) | participant | 244˙503.00 |
| 23 |
UNIVERSITAETSKLINIKUM FREIBURG
Organization address
address: HUGSTETTER STRASSE 49 contact info |
DE (FREIBURG) | participant | 207˙212.60 |
| 24 |
ROYAL HOLLOWAY AND BEDFORD NEW COLLEGE
Organization address
address: EGHAM HILL UNIVERSITY OF LONDON contact info |
UK (EGHAM) | participant | 173˙334.00 |
| 25 |
CENTRO DE INVESTIGACIONES ENERGETICAS, MEDIOAMBIENTALES Y TECNOLOGICAS-CIEMAT
Organization address
address: Avenida Complutense 22 contact info |
ES (MADRID) | participant | 66˙276.00 |
| 26 |
CHEMOTHERAPEUTISCHES FORSCHUNGSINSTITUT GEORG-SPEYER-HAUS STIFTUNG
Organization address
address: PAUL EHRLICH STRASSE 42-44 contact info |
DE (FRANKFURT) | participant | 60˙000.00 |
| 27 |
CHARITE - UNIVERSITAETSMEDIZIN BERLIN
Organization address
address: Chariteplatz 1 contact info |
DE (BERLIN) | participant | 20˙498.16 |
| 28 |
ACIES SAS
Organization address
address: RUE DE LA REPUBLIQUE 69 contact info |
FR (LYON) | participant | 19˙529.19 |
| 29 |
NOVAMEN SAS
Organization address
city: LYON contact info |
FR (LYON) | participant | 0.00 |
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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.