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Advances in Lipid Nanoparticles for siRNA Delivery
Department of Biochemistry and Molecular Biology, University of British Columbia, 2350 Health Sciences Mall, Vancouver, B.C. V6T 1Z3, Canada
* Author to whom correspondence should be addressed.
Received: 1 July 2013; in revised form: 4 September 2013 / Accepted: 12 September 2013 / Published: 18 September 2013
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.
Keywords: lipid nanoparticle; siRNA; ionizable amino lipid; pKa; PEG lipid; targeting
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MDPI and ACS Style
Tam, Y.Y.C.; Chen, S.; Cullis, P.R. Advances in Lipid Nanoparticles for siRNA Delivery. Pharmaceutics 2013, 5, 498-507.
Tam YYC, Chen S, Cullis PR. Advances in Lipid Nanoparticles for siRNA Delivery. Pharmaceutics. 2013; 5(3):498-507.
Tam, Yuen Y.C.; Chen, Sam; Cullis, Pieter R. 2013. "Advances in Lipid Nanoparticles for siRNA Delivery." Pharmaceutics 5, no. 3: 498-507.