作者
Owen S Fenton, Kevin J Kauffman, Rebecca L McClellan, Eric A Appel, J Robert Dorkin, Mark W Tibbitt, Michael W Heartlein, Frank DeRosa, Robert Langer, Daniel G Anderson
发表日期
2016/4/4
期刊
Advanced materials (Deerfield Beach, Fla.)
卷号
28
期号
15
页码范围
2939
出版商
NIH Public Access
简介
Thousands of human diseases could be treated by selectively controlling the expression of specific proteins in vivo. We demonstrate a new series of alkenyl amino alcohol (AAA) ionizable lipids that, when formulated into lipid nanoparticles (LNPs), are capable of delivering human mRNA with unprecedented levels of in vivo efficacy. This study highlights the importance of utilizing synthesis tools in tandem with biological inspiration to understand and improve nucleic acid delivery in vivo.
学术搜索中的文章
OS Fenton, KJ Kauffman, RL McClellan, EA Appel… - Advanced materials (Deerfield Beach, Fla.), 2016