Published online Feb 20, 2015. doi: 10.5493/wjem.v5.i1.11
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: 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.