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NONHUMAN ADENOVIRUS VECTORS FOR GENE TRANSFER TO THE BRAIN

NONHUMAN ADENOVIRUS VECTORS FOR GENE TRANSFER TO THE BRAIN

Objective

Formidable challenges remain to prevent and treat successfully neurodegenerative diseases. Traditional pharmacological approaches, as well as those using stem cells, have made progress but their impact remain limited. As suggested by clinical results in Canavan and Parkinson’s disease, gene transfer offers substantial potential. However, this strategy of therapeutic intervention also brings unique obstacles - in particular the need to address feasibility, efficacy and safety. BrainCAV's foundation is the potential of canine adenovirus type 2 (CAV-2) vectors, which preferentially transduce neurons and undergo a very efficient long-distance targeting via axonal transport. Moreover, the episomal long-term expression leads to safe, efficient neuron-specific gene delivery. We proposed a structured translational approach that spans basic research through pre-clinical model feasibility, efficacy and safety. To provide a proof-of-principle of the effectiveness of CAV-2, we tackle mucopolysaccharidosis type VII, a global, orphan disease commonly affecting children, and Parkinson's disease, a focal degeneration of dopaminergic neurones commonly affecting aged population. To develop and execute this project, BrainCAV brings together an interdisciplinary combination of partners with unique expertise that will take CAV-2 vectors to the doorstep of clinical trials.
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Coordinator

CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE CNRS

Address

Rue Michel Ange 3
75794 Paris

France

Activity type

Higher or Secondary Education Establishments

EU Contribution

€ 433 137,86

Administrative Contact

Jocelyn Mere (Mr.)

Participants (10)

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CANCER RESEARCH UK LBG

United Kingdom

EU Contribution

€ 39 750

UNIVERSITA DEGLI STUDI DI ROMA LA SAPIENZA

Italy

EU Contribution

€ 267 376

INSTITUTO DE BIOLOGIA EXPERIMENTAL E TECNOLOGICA

Portugal

EU Contribution

€ 324 400

UNIVERSITAT AUTONOMA DE BARCELONA

Spain

EU Contribution

€ 254 440

UNIVERSITY OF GLASGOW

United Kingdom

EU Contribution

€ 261 900

FUNDACION PARA LA INVESTIGACION MEDICA APLICADA FIMA

Spain

EU Contribution

€ 268 474

UNIVERSITAET LEIPZIG

Germany

EU Contribution

€ 367 947,14

INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE

France

EU Contribution

€ 356 674

GENIBET - BIOPHARMACEUTICALS SA

Portugal

EU Contribution

€ 252 000

INSERM TRANSFERT SA

France

EU Contribution

€ 158 900

Project information

Grant agreement ID: 222992

Status

Closed project

  • Start date

    1 October 2008

  • End date

    31 March 2013

Funded under:

FP7-HEALTH

  • Overall budget:

    € 4 325 637,23

  • EU contribution

    € 2 984 999

Coordinated by:

CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE CNRS

France

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