2. Academic Validation
  3. Bioinspired Alkenyl Amino Alcohol Ionizable Lipid Materials for Highly Potent In Vivo mRNA Delivery

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

  • Adv Mater. 2016 Apr 20;28(15):2939-43. doi: 10.1002/adma.201505822.
Owen S Fenton 1 2,  Kevin J Kauffman 1 3,  Rebecca L McClellan 1,  Eric A Appel 1,  J Robert Dorkin 1 4,  Mark W Tibbitt 1,  Michael W Heartlein 5,  Frank DeRosa 5,  Robert Langer 1 3 6 7,  Daniel G Anderson 1 3 6 7
Affiliations

Affiliations

  • 1 Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA.
  • 2 Department of Chemistry, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA.
  • 3 Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA.
  • 4 Department of Biology, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA.
  • 5 Shire Pharmaceuticals, Lexington, MA, 02421, USA.
  • 6 Institute for Medical Engineering and Science, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA.
  • 7 Harvard-MIT Division of Health Science and Technology, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA.
PMID: 26889757 DOI: 10.1002/adma.201505822
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.

Keywords

RNA; biomaterials; drug delivery; nanoparticles; proteins.

Figures
Products
  • Cat. No.
    Product Name
    Description
    Target
    Research Area
  • HY-145795
    ≥98.0%, Ionizable Lipid