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 SchemeCSC - Cost-sharing contracts