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

被引：187
作者：
Fenton, Owen S. [1 ,2 ]
Kauffman, Kevin J. [1 ,3 ]
McClellan, Rebecca L. [1 ]
Appel, Eric A. [1 ]
Dorkin, J. Robert [1 ,4 ]
Tibbitt, Mark W. [1 ]
Heartlein, Michael W. [5 ]
DeRosa, Frank [5 ]
Langer, Robert [1 ,3 ,6 ,7 ]
Anderson, Daniel G. [1 ,3 ,6 ,7 ]
机构：
[1] MIT, Koch Inst Integrat Canc Res, 77 Massachusetts Ave, Cambridge, MA 02139 USA
[2] MIT, Dept Chem, Cambridge, MA 02139 USA
[3] MIT, Dept Chem Engn, Cambridge, MA 02139 USA
[4] MIT, Dept Biol, 77 Massachusetts Ave, Cambridge, MA 02139 USA
[5] Shire Pharmaceut, Lexington, MA 02421 USA
[6] MIT, Inst Med Engn & Sci, 77 Massachusetts Ave, Cambridge, MA 02139 USA
[7] MIT, Harvard Mit Div Hlth Sci & Technol, Cambridge, MA 02139 USA
来源：
ADVANCED MATERIALS | 2016年 / 28卷 / 15期
基金：
美国国家科学基金会; 英国惠康基金;
关键词：
biomaterials; drug delivery; nanoparticles; proteins; RNA; SIRNA DELIVERY; ERYTHROPOIETIN; NANOPARTICLES; THERAPEUTICS; EFFICIENCY; MICE;
D O I：
10.1002/adma.201505822
中图分类号：
O6 [化学];
学科分类号：
0703 ;
摘要：
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.
引用
收藏
页码：2939 / 2943
页数：5
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