NOSMOD

Novel Strategies for the Modulation of Immunity to AAV vectors

Coordinatore	UNIVERSITE PIERRE ET MARIE CURIE - PARIS 6    more  Organization address address: Place Jussieu 4 city: PARIS postcode: 75252 contact info Titolo: Mr. Nome: Thomas Cognome: Wiest Email: send email Telefono: 33144279758
Nazionalità Coordinatore	France [FR]
Totale costo	100˙000 €
EC contributo	100˙000 €
Programma	FP7-PEOPLE Specific programme "People" implementing the Seventh Framework Programme of the European Community for research, technological development and demonstration activities (2007 to 2013)
Code Call	FP7-PEOPLE-2012-CIG
Funding Scheme	MC-CIG
Anno di inizio	2013
Periodo (anno-mese-giorno)	2013-05-01   -   2017-04-30

 Partecipanti

#	participant	country	role	EC contrib. [€]
1	UNIVERSITE PIERRE ET MARIE CURIE - PARIS 6  Organization address address: Place Jussieu 4 city: PARIS postcode: 75252 contact info Titolo: Mr. Nome: Thomas Cognome: Wiest Email: send email Telefono: 33144279758	FR (PARIS)	coordinator	100˙000.00

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 Obiettivo del progetto (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.'