Title of project or programme

Nonhuman adenovirus vectors for gene transfer to the brain (BRAINCAV)

Principal Investigators of project/programme grant
Title Forname Surname Institution Country
Dr Eric Kremer CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE France
Address of institution of lead PI
Institution CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE
Street Address
City
Postcode
Country

France

Source of funding information

European Commission

Total sum awarded (Euro)

2984999

Start date of award

01-10-2008

Total duration of award in months

48

The project/programme is most relevant to
  • Parkinson’s disease
Keywords
Research abstract in English

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.

Lay summary

Types: Investments > €500k
Member States: France
Diseases: Parkinson's disease & PD-related disorders
Years: 2011
Database Categories: N/A
Database Tags: N/A

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