RETINAL-GENE-THERAPY

Overcoming translational barriers to gene therapy by novel vector design

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: +33 1 44 27 97 58
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: +33 1 44 27 97 58	FR (PARIS)	coordinator	100˙000.00

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 Obiettivo del progetto (Objective)

'Inherited retinal degenerations are a primary clinical focus of adeno-associated virus (AAV) mediated gene therapy. These diseases mostly involve pathogenic mutations in the retinal pigment epithelium or photoreceptor transcripts leading to cell death and progressively lead to blindness. Current delivery methods are efficient for gene delivery to these cells as well as other therapeutically relevant cell types in the mouse retina. However the applicability of these vectors in human gene therapy depends on our ability to overcome the translational challenges between the mouse and human retinas. Naturally occuring AAVs efficient in transducing the mouse retina in most cases lead to insufficient expression in the primate retina due to interspecies differences in the patterns of transduction by AAV. To address the need for more performant AAVs for retinal gene delivery, we developed artificial AAV variants that have superior tranduction properties to the naturally occuring ones in the mouse retina. A resulting novel AAV variant mediated widespread gene delivery to the outer retina when injected into the vitreous in mice and in some areas of the primate retina. We would like to evaluate the therapeutic potential of this and other AAV variants based on this vector in the context of the primate retina. Our aim is to find suitable variants for application in three different gene therapy settings: gene replacement therapies aimed at the outer retina, trophic factor secretion by overexpression of neurotrophin genes and delivery of optogenetic tools to restrore light sensitivity to blind retinas after loss of vision.'