Maximizing the potency of siRNA lipid nanoparticles for hepatic gene silencing in vivo

…, L Eltepu, S Matsuda, JK Narayanannair…

Index: Jayaraman, Muthusamy; Ansell, Steven M.; Mui, Barbara L.; Tam, Ying K.; Chen, Jianxin; Du, Xinyao; Butler, David; Eltepu, Laxman; Matsuda, Shigeo; Narayanannair, Jayaprakash K.; Rajeev, Kallanthottathil G.; Hafez, Ismail M.; Akinc, Akin; Maier, Martin A.; Tracy, Mark A.; Cullis, Pieter R.; Madden, Thomas D.; Manoharan, Muthiah; Hope, Michael J. Angewandte Chemie - International Edition, 2012 , vol. 51, # 34 p. 8529 - 8533

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Citation Number: 179

Abstract

RNA interference (RNAi) is a ubiquitous and highly effective biological mechanism to control gene expression. Over the last few years it has been established that synthetic doublestranded, small interfering RNA (siRNA) molecules can be applied as therapeutics that potently harness RNAi in humans and animal models, by demonstrating silencing of target mRNA transcripts in rodents and non-human primates (NHP).[1] Because of their ...