LENTIVIRAL VECTORS: Lentiviral vectors for gene therapy of the hematopoietic system: developement from bench to bedside

• Research unit: EU RFP
• Project number: 00.0219

Key words (English)

Gene therapy; stem cells; blood disorders; lentiviral vectors;
Biotechnology; Environmental Protection; Innovation; Technology Transfer; Life Sciences; Medicine; Health; Waste Management

Alternative project number (English)

EU project number: h QLK3-1999-00859

Research programs (English)

EU-programme: 5. Frame Research Programme - 1.1.3 The 'cell factory'

Short description (English)

See abstract

Further information (English)

Full name of research-institution/enterprise:
Université de Genève
Département de Génétique et Microbiologie
CMU

Partners and International Organizations (English)

Coordinator: Unicesità degli Studi di Torino (I)

Abstract (English)

Gene transfer into the hematopoietic system is a promising therapy for several inherited (immunodeficiencies, lysosomal storage disorders, hemophilias) or acquired human diseases (AIDS, cancer). Yet the genetic modification of human hematopoietic stem cells (HSC) to provide a lifelong supply of corrected progeny remain s the most daunting challenge to the success of this endeavor, 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 nondividing cells, including HSC. This project aims at exploiting the tremendous potential of lenti viral 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 f or the competitiveness of the EU biomedical industry.

References in databases (English)

Swiss Database: Euro-DB of the
State Secretariat for Education and Research
Hallwylstrasse 4
CH-3003 Berne, Switzerland
Tel. [phone]
Swiss Project-Number: 00.0219