Copyright ©The Author(s) 2015. Published by Baishideng Publishing Group Inc. All rights reserved.
World J Exp Med. Feb 20, 2015; 5(1): 11-20
Published online Feb 20, 2015. doi: 10.5493/wjem.v5.i1.11
Internal ribosome entry site-based vectors for combined gene therapy
Edith Renaud-Gabardos, Fransky Hantelys, Florent Morfoisse, Xavier Chaufour, Barbara Garmy-Susini, Anne-Catherine Prats
Edith Renaud-Gabardos, Fransky Hantelys, Anne-Catherine Prats, Université de Toulouse, UPS, TRADGENE, EA4554, BP 84225, F-31432 Toulouse, France
Florent Morfoisse, Barbara Garmy-Susini, Inserm, U1048, F-31432 Toulouse, France and Université de Toulouse, UPS, I2MC, F-31432 Toulouse, France
Xavier Chaufour, Centre Hospitalier Universitaire de Toulouse, F-31059 Toulouse and Université de Toulouse, UPS, TRADGENE, EA4554, BP 84225, F-31432 Toulouse, France
Author contributions: Renaud-Gabardos E, Hantelys F, Morfoisse F, Chaufour X, Garmy-Susini B and Prats AC contributed to paper writing.
Conflict-of-interest: The authors declare they have no conflicting interests (including but not limited to commercial, personal, political, intellectual, or religious interests) related to the present work.
Open-Access: This article is an open-access article which was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See:
Correspondence to: Anne-Catherine Prats, PhD, Université de Toulouse, UPS, TRADGENE, EA 4554, I2MC, 1, Avenue Jean Poulhes, BP 84225, 31432 Toulouse cedex 4, F-31432 Toulouse, France.
Telephone: +33-53-1224087 Fax: +33-56-1325622
Received: October 18, 2014
Peer-review started: October 18, 2014
First decision: November 20, 2014
Revised: December 8, 2014
Accepted: December 18, 2014
Article in press: December 19, 2014
Published online: February 20, 2015
Core Tip

Core tip: Combined gene therapy has emerged for a few years as a promising strategy to improve treatments of many diseases including cancer, cardiovascular diseases and degenerative diseases. In this context, internal ribosome entry site (IRES)-based vectors provide a powerful system to co-express several therapeutic genes from the same transcription unit. IRESs are translational enhancers, exhibiting tissue-specificity, and activated by stress. Different IRES-based vectors including plasmids, adeno-associated virus-derived and lentiviral vectors have been used successfully in many preclinical protocols of gene therapy. Moreover the few clinical assays launched with IRES-based multicistronic vectors resulted in therapeutic benefits.