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

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 s...

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Main Authors: DeRosa, Frank, Langer, Robert, Kauffman, Kevin John, McClellan, Rebecca L, Appel, Eric, Dorkin, Joseph Robert, Tibbitt, Mark W, Langer, Robert S, Anderson, Daniel Griffith, Fenton, Owen Shea
Other Authors: Massachusetts Institute of Technology. Institute for Medical Engineering & Science
Format: Article
Language:en_US
Published: Wiley Blackwell 2017
Online Access:http://hdl.handle.net/1721.1/106890
https://orcid.org/0000-0002-5585-9280
https://orcid.org/0000-0002-9436-2453
https://orcid.org/0000-0002-2301-7126
https://orcid.org/0000-0002-4917-7187
https://orcid.org/0000-0003-4255-0492
https://orcid.org/0000-0001-5629-4798
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author DeRosa, Frank
Langer, Robert
Kauffman, Kevin John
McClellan, Rebecca L
Appel, Eric
Dorkin, Joseph Robert
Tibbitt, Mark W
Langer, Robert S
Anderson, Daniel Griffith
Fenton, Owen Shea
author2 Massachusetts Institute of Technology. Institute for Medical Engineering & Science
author_facet Massachusetts Institute of Technology. Institute for Medical Engineering & Science
DeRosa, Frank
Langer, Robert
Kauffman, Kevin John
McClellan, Rebecca L
Appel, Eric
Dorkin, Joseph Robert
Tibbitt, Mark W
Langer, Robert S
Anderson, Daniel Griffith
Fenton, Owen Shea
author_sort DeRosa, Frank
collection MIT
description 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.
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spelling mit-1721.1/1068902022-09-30T13:53:25Z Bioinspired Alkenyl Amino Alcohol Ionizable Lipid Materials for Highly Potent In Vivo mRNA Delivery DeRosa, Frank Langer, Robert Kauffman, Kevin John McClellan, Rebecca L Appel, Eric Dorkin, Joseph Robert Tibbitt, Mark W Langer, Robert S Anderson, Daniel Griffith Fenton, Owen Shea Massachusetts Institute of Technology. Institute for Medical Engineering & Science Harvard University--MIT Division of Health Sciences and Technology Massachusetts Institute of Technology. Department of Biology Massachusetts Institute of Technology. Department of Chemical Engineering Massachusetts Institute of Technology. Department of Chemistry Koch Institute for Integrative Cancer Research at MIT Fenton, Owen S. Kauffman, Kevin John McClellan, Rebecca L Appel, Eric Dorkin, Joseph Robert Tibbitt, Mark W Langer, Robert S Anderson, Daniel Griffith 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. National Science Foundation (U.S.). Graduate Research Fellowship Program Shire Pharmaceuticals Wellcome Trust-MIT Postdoctoral Fellowship 2017-02-10T15:50:07Z 2017-02-10T15:50:07Z 2016-02 2016-01 Article http://purl.org/eprint/type/JournalArticle 0935-9648 1521-4095 http://hdl.handle.net/1721.1/106890 Fenton, Owen S. et al. “Bioinspired Alkenyl Amino Alcohol Ionizable Lipid Materials for Highly Potent In Vivo mRNA Delivery.” Advanced Materials 28.15 (2016): 2939–2943. https://orcid.org/0000-0002-5585-9280 https://orcid.org/0000-0002-9436-2453 https://orcid.org/0000-0002-2301-7126 https://orcid.org/0000-0002-4917-7187 https://orcid.org/0000-0003-4255-0492 https://orcid.org/0000-0001-5629-4798 en_US http://dx.doi.org/10.1002/adma.201505822 Advanced Materials Creative Commons Attribution-Noncommercial-Share Alike http://creativecommons.org/licenses/by-nc-sa/4.0/ application/pdf Wiley Blackwell PMC
spellingShingle DeRosa, Frank
Langer, Robert
Kauffman, Kevin John
McClellan, Rebecca L
Appel, Eric
Dorkin, Joseph Robert
Tibbitt, Mark W
Langer, Robert S
Anderson, Daniel Griffith
Fenton, Owen Shea
Bioinspired Alkenyl Amino Alcohol Ionizable Lipid Materials for Highly Potent In Vivo mRNA Delivery
title Bioinspired Alkenyl Amino Alcohol Ionizable Lipid Materials for Highly Potent In Vivo mRNA Delivery
title_full Bioinspired Alkenyl Amino Alcohol Ionizable Lipid Materials for Highly Potent In Vivo mRNA Delivery
title_fullStr Bioinspired Alkenyl Amino Alcohol Ionizable Lipid Materials for Highly Potent In Vivo mRNA Delivery
title_full_unstemmed Bioinspired Alkenyl Amino Alcohol Ionizable Lipid Materials for Highly Potent In Vivo mRNA Delivery
title_short Bioinspired Alkenyl Amino Alcohol Ionizable Lipid Materials for Highly Potent In Vivo mRNA Delivery
title_sort bioinspired alkenyl amino alcohol ionizable lipid materials for highly potent in vivo mrna delivery
url http://hdl.handle.net/1721.1/106890
https://orcid.org/0000-0002-5585-9280
https://orcid.org/0000-0002-9436-2453
https://orcid.org/0000-0002-2301-7126
https://orcid.org/0000-0002-4917-7187
https://orcid.org/0000-0003-4255-0492
https://orcid.org/0000-0001-5629-4798
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