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1. Bioinspired Alkenyl Amino Alcohol Ionizable Lipid Materials for Highly Potent In Vivo mRNA Delivery.

Author

Fenton OS, Kauffman KJ, McClellan RL, Appel EA, Dorkin JR, Tibbitt MW, Heartlein MW, DeRosa F, Langer R, and Anderson DG

Subjects
Erythropoietin genetics, Humans, RNA, Messenger chemistry, RNA, Messenger genetics, Alkenes chemistry, Amino Alcohols chemistry, Biomimetic Materials chemistry, Drug Carriers chemistry, Lipids chemistry, Nanoparticles chemistry
Abstract

Thousands of human diseases could be treated by selectively controlling the expression of specific proteins in vivo. A new series of alkenyl amino alcohol (AAA) ionizable lipid nanoparticles (LNPs) capable of delivering human mRNA with unprecedented levels of in vivo efficacy is demonstrated. This study highlights the importance of utilizing synthesis tools in tandem with biological inspiration to understand and improve nucleic acid delivery in vivo., (© 2016 WILEY-VCH Verlag GmbH & Co. KGaA, Weinheim.)

Published
2016
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