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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.

Field of science

  • /medical and health sciences/medical biotechnology/cells technologies/stem cells
  • /medical and health sciences/basic medicine/neurology/parkinson
  • /medical and health sciences/medical biotechnology/genetic engineering/gene therapy
  • /social sciences/social and economic geography/transport

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

CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE CNRS
Address
Rue Michel Ange 3
75794 Paris
France
Activity type
Research Organisations
EU contribution
€ 433 137,86
Administrative Contact
Jocelyn Mere (Mr.)

Participants (10)

CANCER RESEARCH UK LBG
United Kingdom
EU contribution
€ 39 750
Address
St John Street 407 Angel Building
EC1V 4AD London
Activity type
Research Organisations
Administrative Contact
Robina Ibanda (Ms.)
UNIVERSITA DEGLI STUDI DI ROMA LA SAPIENZA
Italy
EU contribution
€ 267 376
Address
Piazzale Aldo Moro 5
00185 Roma
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Luigina Travaglini (Ms.)
INSTITUTO DE BIOLOGIA EXPERIMENTAL E TECNOLOGICA
Portugal
EU contribution
€ 324 400
Address
Avenida Da Republica Quinta Do Marques
2781-901 Oeiras
Activity type
Research Organisations
Administrative Contact
Elsa Vieira (Dr.)
UNIVERSIDAD AUTONOMA DE BARCELONA
Spain
EU contribution
€ 254 440
Address
Calle Campus Universitario Sn Cerdanyola V
08290 Cerdanyola Del Valles
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Dirk Zahn (Dr.)
UNIVERSITY OF GLASGOW
United Kingdom
EU contribution
€ 261 900
Address
University Avenue
G12 8QQ Glasgow
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Joe Galloway (Mr.)
FUNDACION PARA LA INVESTIGACION MEDICA APLICADA FIMA
Spain
EU contribution
€ 268 474
Address
Avenida De Pio Xii 55
31008 Pamplona
Activity type
Research Organisations
Administrative Contact
Andrew Solomon (Dr.)
UNIVERSITAET LEIPZIG

Participation ended

Germany
EU contribution
€ 367 947,14
Address
Ritterstrasse 26
04109 Leipzig
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Sabine Herden (Dr.)
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
France
EU contribution
€ 356 674
Address
Rue De Tolbiac 101
75654 Paris
Activity type
Research Organisations
Administrative Contact
Emmanuel Berthenand (Mr.)
GENIBET - BIOPHARMACEUTICALS SA
Portugal
EU contribution
€ 252 000
Address
Avenida Da Republica Quinta Do
2781 901 Oeiras
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
António Duarte (Prof.)
INSERM TRANSFERT SA
France
EU contribution
€ 158 900
Address
Rue Watt 7
75013 Paris
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Louis Jammayrac (Mr.)