title: Bioinspired Alkenyl Amino Alcohol Ionizable Lipid Materials for Highly Potent In Vivo mRNA Delivery.
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.
authors.affiliation: Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA.|Department of Chemistry, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA.
authors.forename: Owen S
authors.initials: OS
authors.lastname: Fenton
authors.affiliation: Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA.|Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA.
authors.forename: Kevin J
authors.initials: KJ
authors.lastname: Kauffman
authors.affiliation: Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA.
authors.forename: Rebecca L
authors.initials: RL
authors.lastname: McClellan
authors.affiliation: Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA.
authors.forename: Eric A
authors.initials: EA
authors.lastname: Appel
authors.affiliation: Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA.|Department of Biology, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA.
authors.forename: J Robert
authors.initials: JR
authors.lastname: Dorkin
authors.affiliation: Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA.
authors.forename: Mark W
authors.initials: MW
authors.lastname: Tibbitt
authors.affiliation: Shire Pharmaceuticals, Lexington, MA, 02421, USA.
authors.forename: Michael W
authors.initials: MW
authors.lastname: Heartlein
authors.affiliation: Shire Pharmaceuticals, Lexington, MA, 02421, USA.
authors.forename: Frank
authors.initials: F
authors.lastname: DeRosa
authors.affiliation: Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA.|Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA.|Institute for Medical Engineering and Science, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA.|Harvard-MIT Division of Health Science and Technology, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA.
authors.forename: Robert
authors.initials: R
authors.lastname: Langer
authors.affiliation: Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA.|Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA.|Institute for Medical Engineering and Science, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA.|Harvard-MIT Division of Health Science and Technology, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA.
authors.forename: Daniel G
authors.initials: DG
authors.lastname: Anderson
mesh_terms: D000475:Alkenes / Q000737:chemistry*; D000605:Amino Alcohols / Q000737:chemistry*; D040761:Biomimetic Materials / Q000737:chemistry*; D004337:Drug Carriers / Q000737:chemistry*; D004921:Erythropoietin / Q000235:genetics; D006801:Humans; D008055:Lipids / Q000737:chemistry*; D053758:Nanoparticles / Q000737:chemistry*; D012333:RNA, Messenger / Q000737:chemistry / Q000235:genetics
chemical_list: D000475:Alkenes; D000605:Amino Alcohols; D004337:Drug Carriers; D008055:Lipids; D012333:RNA, Messenger; D004921:Erythropoietin
keywords: RNA; biomaterials; drug delivery; nanoparticles; proteins