Objective
Adeno-associated virus (AAV) vectors are among the most efficient vectors for in vivo gene transfer. Experience in human gene transfer trials showed that targeting hepatocytes with AAV vectors has the potential to correct diseases like hemophilia, congenital blindness, and lipoprotein lipase deficiency. However, these studies also suggest that one of the last obstacles to be overcome in order to achieve long-term disease correction is the immune system. As a result of exposure to wild type AAV, a significant portion of the human population will develop both humoral and cellular immunity to the virus, which can greatly affect the safety and efficacy of gene transfer with recombinant AAV vectors. This proposal combines knowledge on immunology and AAV vector mediated gene transfer, with the ultimate goal of developing safe and effective gene therapy protocols. Studies in humans undergoing AAV gene transfer will help identify markers of T cell activation and correlate them with the outcome of gene transfer. Studies in humans receiving immunosuppressive (IS) regimens will help identifying drug regimens that could be safely used to modulate B and T cell immunity to AAV in the context of gene transfer. In the third aim of this proposal, IS regimens will be tested in vivo in the relevant animal models. Results from these studies will contribute to design safe and effective clinical gene transfer studies by providing new tools and markers for safety assessment and early intervention aimed at modulating detrimental immune responses directed against the AAV vector capsid. Furthermore, these studies address the issue of humoral immunity to AAV by identifying strategies to overcome the limitation of anti-AAV antibodies, thus allowing for treatment of a significant proportion of humans otherwise non eligible for enrollment in gene transfer trials.
Fields of science (EuroSciVoc)
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
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Programme(s)
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
Topic(s)
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Call for proposal
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
FP7-PEOPLE-2012-CIG
See other projects for this call
Funding Scheme
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
MC-CIG - Support for training and career development of researcher (CIG)
Coordinator
75252 PARIS
France
The total costs incurred by this organisation to participate in the project, including direct and indirect costs. This amount is a subset of the overall project budget.