Introduction: Manipulating gene activity represents a promising approach for the treatment of cancer and other diseases. The relatively recent discovery of RNA interference (RNAi) revolutionized therapeutic approaches in this field. RNA effectors can now be used to modify the activity of genes and theoretically control any biological process.
Area covered: However, the clinical application of RNAi has been limited by the inefficient delivery of RNA. Challenges associated with the in vivo use of RNAi mediators, include rapid degradation, uptake by the reticular endothelial system and inefficient cellular internalization. To date, various strategies have been developed in order to overcome these pitfalls. Among these approaches, non-viral delivery systems have gained increasing popularity, as they are generally considered safer than their viral counterparts.
Expert opinion: The use of cationic polymers, especially polyethylenimine and chitosan, for the in vivo delivery of doubled-stranded RNAs is discussed in this review.