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Lentiviral vectors for gene therapy of the hematopoietic system: development from bench to bedside

Objective

Gene transfer into the haematopoietic system is a promising therapy for several inherited (immunodeficiency’s, liposome storage disorders, haemophilias) or acquired human diseases (AIDS, cancer). Yet the genetic modification of human haematopoietic stem cells (HSC) to provide a lifelong supply of corrected progeny remains the most daunting challenge to the success of this endeavour, because HSC are mostly quiescent and hence difficult to transduce. A major breakthrough has been obtained through the creation of lentiviral vectors, which can mediate the efficient integration and long-term expression of transgenes into no dividing cells, including HSC. This project aims at exploiting the tremendous potential of lentiviral vectors for gene transfer into HSC, in order to bring this new technology from bench to bedside for the therapy of human disease, and to exact its value for the competitiveness of the EU biomedical industry.

Funding Scheme

CSC - Cost-sharing contracts

Coordinator

UNIVERSITY OF TORINO
Address
Via Giuseppe Verdi 8
10124 Torino (Turin)
Italy

Participants (5)

FONDAZIONE CENTRO SAN RAFFAELE DEL MONTE TABOR - ISTITUTO DI RICOVERO E CURA A CARATTERE SCIENTIFICO
Italy
Address
Via Olgettina 58
20132 Milano
GENETHON III
France
Address
Rue De L'internationale 1Bis
91002 Evry
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
France
Address
46,Allée D'italie 46 Adr 5 - Rhône-alpes, Auvergne, B
69364 Lyon
MOLECULAR MEDICINE SPA
Italy
Address
Via Olgettina 58
Milano
UNIVERSITY OF GENEVA
Switzerland
Address
24,Rue General Dufour 24
1211 Geneve