Open Access

siRNA delivery: from basics to therapeutic applications

Tiziana Musacchio1, Vladimir P. Torchilin1,*
Center for Pharmaceutical Biotechnology and Nanomedicine, Northeastern University, 360 Huntington Ave, 02115, Boston, MA, USA
DOI: 10.2741/4087 Volume 18 Issue 1, pp.58-79
Published: 01 January 2013
*Corresponding Author(s):  
Vladimir P. Torchilin

The chance to selectively intervene and stop the development of any gene-dependent disease in different organs and pathologies makes siRNA an ideal therapeutic agent. However, serious issues should be addressed before the real therapeutic use of siRNA. The poor pharmacokinetic properties of siRNA, its short half-life, its low in vivo stability, its fast elimination by kidney filtration and its low transfection efficiency complicate the use of siRNA as a therapeutic molecule. In this review, we will describe the latest and most advanced approaches and strategies undertaken to address these limitations and improve siRNA delivery and its gene silencing efficacy as well as the prospects for its therapeutic applications.

Key words

siRNA, siRNA conjugate, siRNA delivery systems, Bioconjugates, Micelles, Liposomes, Carbonanotubes, Nanogels, Dendrimers, Lipid-based- particles, Polymer-based-particles, Clinical trials, Review

Share and Cite
Tiziana Musacchio, Vladimir P. Torchilin. siRNA delivery: from basics to therapeutic applications. Frontiers in Bioscience-Landmark. 2013. 18(1); 58-79.