Advances in Lipid Nanoparticles for siRNA Delivery

Pharmaceutics

Yuen Yi C. Tam¹  Sam Chen² 

[1] Department of Biochemistry and Molecular Biology, University of British Columbia, 2350 Health Sciences Mall, Vancouver, B.C. V6T 1Z3, Canada;

关键词: lipid nanoparticle;    siRNA;    ionizable amino lipid;    pKa;    PEG lipid;    targeting;   

DOI  :  10.3390/pharmaceutics5030498

来源: mdpi

【 摘 要 】

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.

【 授权许可】

CC BY   
© 2013 by the authors; licensee MDPI, Basel, Switzerland.