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

• Unité de recherche: PCRD EU
• Numéro de projet: 00.0219
• Titre du projet anglais: LENTIVIRAL VECTORS: Lentiviral vectors for gene therapy of the hematopoietic system: developement from bench to bedside

Mots-clé (Anglais)

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

Autre Numéro de projet (Anglais)

EU project number: h QLK3-1999-00859

Programme de recherche (Anglais)

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

Description succincte (Anglais)

See abstract

Autres indications (Anglais)

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

Partenaires et organisations internationales (Anglais)

Coordinator: Unicesità degli Studi di Torino (I)

Résumé des résultats (Abstract) (Anglais)

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.

Références bases de données (Anglais)

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