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. 2013 Sep 18;5(3):498-507.
doi: 10.3390/pharmaceutics5030498.

Advances in Lipid Nanoparticles for siRNA Delivery

Yuen Yi C Tam  1 , Sam Chen, Pieter R Cullis
Affiliations

Advances in Lipid Nanoparticles for siRNA Delivery

Yuen Yi C Tam et al. Pharmaceutics. .

Abstract

Technological advances in both siRNA (small interfering RNA) and whole genome sequencing have demonstrated great potential in translating genetic information into siRNA-based drugs to halt the synthesis of most disease-causing proteins. Despite its powerful promises as a drug, siRNA requires a sophisticated delivery vehicle because of its rapid degradation in the circulation, inefficient accumulation in target tissues and inability to cross cell membranes to access the cytoplasm where it functions. Lipid nanoparticle (LNP) containing ionizable amino lipids is the leading delivery technology for siRNA, with five products in clinical trials and more in the pipeline. Here, we focus on the technological advances behind these potent systems for siRNA-mediated gene silencing.

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Figures

Figure 1
Figure 1
Lipid nanoparticle (LNP) siRNA systems are typically composed of ionizable amino lipids, phosphatidylcholine lipids, cholesterol and polyethylene glycol-lipid conjugate (PEG-lipids). Structure of heptatriaconta-6,9,28,31-tetraen-19-yl 4-(dimethylamino)butanoate (DLin-MC3-DMA), 1,2-distearoyl-sn-glycero-3-phosphocholine (DSPC), polyethylene glycol-dimyristolglycerol (PEG-DMG) and cholesterol is shown.
Figure 2
Figure 2
A schematic of LNP siRNA showing a nanostructured core.
See this image and copyright information in PMC

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