Literature DB >> 26889757

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

Owen S Fenton1,2, Kevin J Kauffman1,3, Rebecca L McClellan1, Eric A Appel1, J Robert Dorkin1,4, Mark W Tibbitt1, Michael W Heartlein5, Frank DeRosa5, Robert Langer1,3,6,7, Daniel G Anderson1,3,6,7.   

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.
© 2016 WILEY-VCH Verlag GmbH & Co. KGaA, Weinheim.

Entities:  

Keywords:  RNA; biomaterials; drug delivery; nanoparticles; proteins

Mesh：

Substances：

Year:  2016        PMID： 26889757      PMCID： PMC5245883          DOI： 10.1002/adma.201505822

Source DB:  PubMed          Journal:  Adv Mater        ISSN： 0935-9648            Impact factor:   30.849


  19 in total

Review 1.  Protein therapeutics: a summary and pharmacological classification.

Authors:  Benjamin Leader; Quentin J Baca; David E Golan
Journal:  Nat Rev Drug Discov       Date:  2008-01       Impact factor: 84.694

2.  Lipid-like materials for low-dose, in vivo gene silencing.

Authors:  Kevin T Love; Kerry P Mahon; Christopher G Levins; Kathryn A Whitehead; William Querbes; J Robert Dorkin; June Qin; William Cantley; Liu Liang Qin; Timothy Racie; Maria Frank-Kamenetsky; Ka Ning Yip; Rene Alvarez; Dinah W Y Sah; Antonin de Fougerolles; Kevin Fitzgerald; Victor Koteliansky; Akin Akinc; Robert Langer; Daniel G Anderson
Journal:  Proc Natl Acad Sci U S A       Date:  2010-01-11       Impact factor: 11.205

Review 3.  Liposomal drug delivery systems: from concept to clinical applications.

Authors:  Theresa M Allen; Pieter R Cullis
Journal:  Adv Drug Deliv Rev       Date:  2012-10-01       Impact factor: 15.470

Review 4.  mRNA-based therapeutics--developing a new class of drugs.

Authors:  Ugur Sahin; Katalin Karikó; Özlem Türeci
Journal:  Nat Rev Drug Discov       Date:  2014-09-19       Impact factor: 84.694

5.  Cationic lipid saturation influences intracellular delivery of encapsulated nucleic acids.

Authors:  James Heyes; Lorne Palmer; Kaz Bremner; Ian MacLachlan
Journal:  J Control Release       Date:  2005-10-03       Impact factor: 9.776

6.  Expression of therapeutic proteins after delivery of chemically modified mRNA in mice.

Authors:  Michael S D Kormann; Günther Hasenpusch; Manish K Aneja; Gabriela Nica; Andreas W Flemmer; Susanne Herber-Jonat; Marceline Huppmann; Lauren E Mays; Marta Illenyi; Andrea Schams; Matthias Griese; Iris Bittmann; Rupert Handgretinger; Dominik Hartl; Joseph Rosenecker; Carsten Rudolph
Journal:  Nat Biotechnol       Date:  2011-01-09       Impact factor: 54.908

Review 7.  Bioactive sphingolipids: metabolism and function.

Authors:  Nana Bartke; Yusuf A Hannun
Journal:  J Lipid Res       Date:  2008-11-17       Impact factor: 5.922

8.  Increased erythropoiesis in mice injected with submicrogram quantities of pseudouridine-containing mRNA encoding erythropoietin.

Authors:  Katalin Karikó; Hiromi Muramatsu; Jason M Keller; Drew Weissman
Journal:  Mol Ther       Date:  2012-02-14       Impact factor: 11.454

9.  Efficiency of siRNA delivery by lipid nanoparticles is limited by endocytic recycling.

Authors:  Gaurav Sahay; William Querbes; Christopher Alabi; Ahmed Eltoukhy; Sovan Sarkar; Christopher Zurenko; Emmanouil Karagiannis; Kevin Love; Delai Chen; Roberto Zoncu; Yosef Buganim; Avi Schroeder; Robert Langer; Daniel G Anderson
Journal:  Nat Biotechnol       Date:  2013-06-23       Impact factor: 54.908

10.  A novel mechanism is involved in cationic lipid-mediated functional siRNA delivery.

Authors:  James J Lu; Robert Langer; Jianzhu Chen
Journal:  Mol Pharm       Date:  2009 May-Jun       Impact factor: 4.939
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  60 in total

Review 1.  Advances in Biomaterials for Drug Delivery.

Authors:  Owen S Fenton; Katy N Olafson; Padmini S Pillai; Michael J Mitchell; Robert Langer
Journal:  Adv Mater       Date:  2018-05-07       Impact factor: 30.849

Review 2.  Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery.

Authors:  Piotr S Kowalski; Arnab Rudra; Lei Miao; Daniel G Anderson
Journal:  Mol Ther       Date:  2019-02-19       Impact factor: 11.454

3.  Preparation and Optimization of Lipid-Like Nanoparticles for mRNA Delivery.

Authors:  Bin Li; Yizhou Dong
Journal:  Methods Mol Biol       Date:  2017

Review 4.  Nanotechnologies in delivery of mRNA therapeutics using nonviral vector-based delivery systems.

Authors:  S Guan; J Rosenecker
Journal:  Gene Ther       Date:  2017-01-17       Impact factor: 5.250

5.  Charge-altering releasable transporters (CARTs) for the delivery and release of mRNA in living animals.

Authors:  Colin J McKinlay; Jessica R Vargas; Timothy R Blake; Jonathan W Hardy; Masamitsu Kanada; Christopher H Contag; Paul A Wender; Robert M Waymouth
Journal:  Proc Natl Acad Sci U S A       Date:  2017-01-09       Impact factor: 11.205

Review 6.  Transfection by cationic gemini lipids and surfactants.

Authors:  M Damen; A J J Groenen; S F M van Dongen; R J M Nolte; B J Scholte; M C Feiters
Journal:  Medchemcomm       Date:  2018-07-17       Impact factor: 3.597

7.  A Potent Branched-Tail Lipid Nanoparticle Enables Multiplexed mRNA Delivery and Gene Editing In Vivo.

Authors:  Khalid A Hajj; Jilian R Melamed; Namit Chaudhary; Nicholas G Lamson; Rebecca L Ball; Saigopalakrishna S Yerneni; Kathryn A Whitehead
Journal:  Nano Lett       Date:  2020-06-09       Impact factor: 11.189

8.  mRNA Therapy Improves Metabolic and Behavioral Abnormalities in a Murine Model of Citrin Deficiency.

Authors:  Jingsong Cao; Ding An; Mikel Galduroz; Jenny Zhuo; Shi Liang; Marianne Eybye; Andrea Frassetto; Eishi Kuroda; Aki Funahashi; Jordan Santana; Cosmin Mihai; Kerry E Benenato; E Sathyajith Kumarasinghe; Staci Sabnis; Timothy Salerno; Kimberly Coughlan; Edward J Miracco; Becca Levy; Gilles Besin; Joshua Schultz; Christine Lukacs; Lin Guey; Patrick Finn; Tatsuhiko Furukawa; Paloma H Giangrande; Takeyori Saheki; Paolo G V Martini
Journal:  Mol Ther       Date:  2019-04-23       Impact factor: 11.454

Review 9.  Nanoscale platforms for messenger RNA delivery.

Authors:  Bin Li; Xinfu Zhang; Yizhou Dong
Journal:  Wiley Interdiscip Rev Nanomed Nanobiotechnol       Date:  2018-05-04

10.  Polymer-Lipid Nanoparticles for Systemic Delivery of mRNA to the Lungs.

Authors:  James C Kaczmarek; Asha K Patel; Kevin J Kauffman; Owen S Fenton; Matthew J Webber; Michael W Heartlein; Frank DeRosa; Daniel G Anderson
Journal:  Angew Chem Int Ed Engl       Date:  2016-09-30       Impact factor: 15.336
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