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

Year: 2016

Type: article

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.

Source: Advanced Materials

DOI: 10.1002/adma.201505822

Authors: 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

Institutions: Koch Institute for Integrative Cancer Research At MIT, Massachusetts Institute of Technology, Shire (United States), Harvard–MIT Division of Health Sciences and Technology

Language: English

Topic: RNA Interference and Gene Delivery

Subfield: Molecular Biology

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: green

Funders: National Science Foundation, Wellcome Trust, Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Shire, National Cancer Institute

Award: Cancer Center Support (Core) Grant

OpenAlex ID: W2281696593

FWCI: 10.57

Cites: 27

Cited by: 252

Related to: 10