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Advanced gene therapy tools for treatment of CNS-specific disorders

Objective

Curative therapies still do not exist for most CNS diseases but gene therapy is a promising new approach. We propose that it will be possible to modify brain function and pathophysiology by targeted delivery of specific curative factors to selected populations of brain cells that are affected by disease. This opens the door for effective treatment regimes, which can be tailored to individual patients needs. However, currently available gene transfer vectors have limitations regarding safety and efficacy, as they do not allow for targeting of specific populations of neurons or glia or regulation of transgene expression. The NEUGENE consortium has been founded by leading European scientists from academia and industry to overcome these limitations. The consortium will develop Adeno-associated virus (AAV) and Lentivirus (LV)- based tools for targeted and regulated gene transfer into different populations of CNS cells. The consortium will provide a selection of vectors that are optimized for different therapeutic approaches, e.g. regulated expression of neurotrophic factors or manipulation of neurotransmitter synthesis in specific neurons. NEUGENE has three major goals:1) targeting gene transfer vectors to specific populations of neurons and glia, by transcriptional regulation and miRNA-mediated de-targeting and by exploiting the cell-specific tropism of novel types of viral vectors, 2) tight control over expression levels of therapeutic genes by using regulated systems based on different principles, and 3) establishing the safety of the novel vector tools. NEUGENE will verify the functional efficacy of the novel CNS gene transfer tools in a well-established animal model of Parkinson's Disease (PD). This disorder affects over 1.000.000 Europeans and is increasing in prevalence with the aging population. Importantly, principles and mechanisms developed and evaluated within the consortium will also be of direct relevance for gene therapy of many other brain disorder.

Field of science

  • /medical and health sciences/basic medicine/physiology/pathophysiology
  • /medical and health sciences/basic medicine/neurology/parkinson
  • /medical and health sciences/medical biotechnology/genetic engineering/gene therapy

Call for proposal

FP7-HEALTH-2007-B
See other projects for this call

Funding Scheme

CP-FP - Small or medium-scale focused research project

Coordinator

UNIVERSITAETSMEDIZIN GOETTINGEN - GEORG-AUGUST-UNIVERSITAET GOETTINGEN - STIFTUNG OEFFENTLICHEN RECHTS
Address
Robert-koch-strasse 40
37075 Goettingen
Germany
Activity type
Higher or Secondary Education Establishments
EU contribution
€ 690 800
Administrative Contact
Christiane Hennecke (Ms.)

Participants (5)

MAX IV Laboratory, Lund University
Sweden
EU contribution
€ 700 800
Address
Paradisgatan 5C
22100 Lund
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Eva Nordin (Ms.)
ROYAL HOLLOWAY AND BEDFORD NEW COLLEGE
United Kingdom
EU contribution
€ 300 000
Address
Egham Hill University Of London
TW20 0EX Egham
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Jenny Febry (Ms.)
COMMISSARIAT A L ENERGIE ATOMIQUE ET AUX ENERGIES ALTERNATIVES
France
EU contribution
€ 756 000
Address
Rue Leblanc 25
75015 Paris 15
Activity type
Research Organisations
Administrative Contact
Olivier Leroy (Mr.)
ECOLE POLYTECHNIQUE FEDERALE DE LAUSANNE
Switzerland
EU contribution
€ 452 400
Address
Batiment Ce 3316 Station 1
1015 Lausanne
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Patrick Aebischer (Prof.)
OXFORD BIOMEDICA (UK) LIMITED
United Kingdom
EU contribution
€ 100 000
Address
Oxford Science Park Medawar
OX4 4GA Oxford
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Peter Nolan (Mr.)