Lipid-based vectors for siRNA delivery

Abstract

siRNA therapeutics has developed rapidly and already there are clinical trials ongoing or planned; however, the delivery of siRNA into cells, tissues or organs remains to be a major obstacle. Lipid-based vectors hold the most promising position among non-viral vectors, as they have a similar structure to cell or organelle membranes. But when used in the form of liposomes, these vectors have shown some problems. Therefore, either the nature of lipids themselves or forms used should be improved. As a novel class of lipid like materials, lipidoids have the advantages of easy synthesis and the ability for delivering siRNA to obtain excellent silencing activity. However, the toxicities of lipidoids have not been thoroughly studied. pH responsive lipids have also gained great attention recently, though some of the amine-based lipids are not novel in terms of chemical structures. More complex self-assembly structures, such as LPD (LPH) and LCP, may provide a good solution to siRNA delivery. They have demonstrated controlled particle morphology and size and siRNA delivery activity for both in vitro and in vivo.

Keywords::

• Lipids
• siRNA delivery
• RNA interference
• gene therapy

Acknowledgement

We would like to express thanks to Kelly Racette for editing the manuscript.

Declaration of interest

The original work was supported by NIH grants (CA129835, CA129421, CA149363 and CA151652), the National Natural Science Foundation of China (21176046) and the Fundamental Research Funds for the Central Universities (DC12010104).