title: Internal ribosome entry site-based vectors for combined gene therapy.
abstract: Gene therapy appears as a promising strategy to treat incurable diseases. In particular, combined gene therapy has shown improved therapeutic efficiency. Internal ribosome entry sites (IRESs), RNA elements naturally present in the 5' untranslated regions of a few mRNAs, constitute a powerful tool to co-express several genes of interest. IRESs are translational enhancers allowing the translational machinery to start protein synthesis by internal initiation. This feature allowed the design of multi-cistronic vectors expressing several genes from a single mRNA. IRESs exhibit tissue specificity, and drive translation in stress conditions when the global cell translation is blocked, which renders them useful for gene transfer in hypoxic conditions occurring in ischemic diseases and cancer. IRES-based viral and non viral vectors have been used successfully in preclinical and clinical assays of combined gene therapy and resulted in therapeutic benefits for various pathologies including cancers, cardiovascular diseases and degenerative diseases.
authors.affiliation: Edith Renaud-Gabardos, Fransky Hantelys, Anne-Catherine Prats, Université de Toulouse, UPS, TRADGENE, EA4554, BP 84225, F-31432 Toulouse, France.
authors.forename: Edith
authors.initials: E
authors.lastname: Renaud-Gabardos
authors.affiliation: Edith Renaud-Gabardos, Fransky Hantelys, Anne-Catherine Prats, Université de Toulouse, UPS, TRADGENE, EA4554, BP 84225, F-31432 Toulouse, France.
authors.forename: Fransky
authors.initials: F
authors.lastname: Hantelys
authors.affiliation: Edith Renaud-Gabardos, Fransky Hantelys, Anne-Catherine Prats, Université de Toulouse, UPS, TRADGENE, EA4554, BP 84225, F-31432 Toulouse, France.
authors.forename: Florent
authors.initials: F
authors.lastname: Morfoisse
authors.affiliation: Edith Renaud-Gabardos, Fransky Hantelys, Anne-Catherine Prats, Université de Toulouse, UPS, TRADGENE, EA4554, BP 84225, F-31432 Toulouse, France.
authors.forename: Xavier
authors.initials: X
authors.lastname: Chaufour
authors.affiliation: Edith Renaud-Gabardos, Fransky Hantelys, Anne-Catherine Prats, Université de Toulouse, UPS, TRADGENE, EA4554, BP 84225, F-31432 Toulouse, France.
authors.forename: Barbara
authors.initials: B
authors.lastname: Garmy-Susini
authors.affiliation: Edith Renaud-Gabardos, Fransky Hantelys, Anne-Catherine Prats, Université de Toulouse, UPS, TRADGENE, EA4554, BP 84225, F-31432 Toulouse, France.
authors.forename: Anne-Catherine
authors.initials: AC
authors.lastname: Prats
keywords: Gene therapy; Gene transfer; Internal ribosome entry site; Vector