Bioinspired Alkenyl Amino Alcohol Ionizable Lipid Materials for Highly Potent In Vivo mRNA Delivery.
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ABSTRACT:
SUBMITTER: Fenton OS
PROVIDER: S-EPMC5245883 | biostudies-literature | 2016 Apr
REPOSITORIES: biostudies-literature
Publications
Bioinspired Alkenyl Amino Alcohol Ionizable Lipid Materials for Highly Potent In Vivo mRNA Delivery.
Fenton Owen S OS Kauffman Kevin J KJ McClellan Rebecca L RL Appel Eric A EA Dorkin J Robert JR Tibbitt Mark W MW Heartlein Michael W MW DeRosa Frank F Langer Robert R Anderson Daniel G DG
Advanced materials (Deerfield Beach, Fla.) 20160218 15
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. ...[more]
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