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

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 s...

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Veröffentlicht in:	Advanced materials (Weinheim) 2016-04, Vol.28 (15), p.2939-2943
Hauptverfasser:	Fenton, Owen S., Kauffman, Kevin J., McClellan, Rebecca L., Appel, Eric A., Dorkin, J. Robert, Tibbitt, Mark W., Heartlein, Michael W., DeRosa, Frank, Langer, Robert, Anderson, Daniel G.
Format:	Artikel
Sprache:	eng
Schlagworte:	Alcohols Alkenes - chemistry Amino Alcohols - chemistry biomaterials Biomimetic Materials - chemistry Drug Carriers - chemistry drug delivery Effectiveness Erythropoietin - genetics Human Humans In vivo methods and tests Inspiration Lipids Lipids - chemistry Materials selection Nanoparticles Nanoparticles - chemistry proteins RNA RNA, Messenger - chemistry RNA, Messenger - genetics
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Beschreibung
Zusammenfassung:	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.
ISSN:	0935-9648 1521-4095
DOI:	10.1002/adma.201505822