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Literature DB >> 28730441

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

Abstract

Lipid-like nanoparticles (LLNs) have shown great promise for nucleic acid delivery. Recently, we have developed N 1,N 3,N 5-tris(2-aminoethyl)benzene-1,3,5-tricarboxamide (TT) derived lipid-like compounds, formulated them into TT LLNs for mRNA delivery, and applied an orthogonal array design to facilitate formulation optimization. This chapter focuses on the following contents relevant to lipid-like nanoparticles: formulation method, particle characterization, orthogonal array design, and in vitro assays.

Entities: CellLine Chemical Disease Gene Mutation Species

Keywords: Delivery efficiency; Lipid-like nanoparticles (LLNs); Optimization; Orthogonal array design; mRNA delivery

Mesh：

Substances：

Year: 2017 PMID： 28730441 PMCID： PMC5921050 DOI： 10.1007/978-1-4939-7138-1_13

Source DB: PubMed Journal: Methods Mol Biol ISSN： 1064-3745

35 in total

1. 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

2. Report: Optimization study of the preparation factors for argan oil microcapsule based on hybrid-level orthogonal array design via SPSS modeling.

Authors: Xi Zhao; Xiaoli Wu; Hui Zhou; Tao Jiang; Chun Chen; Mingshi Liu; Yuanbao Jin; Dongsheng Yang
Journal: Pak J Pharm Sci Date: 2014-11 Impact factor: 0.684

3. Lipopeptide nanoparticles for potent and selective siRNA delivery in rodents and nonhuman primates.

Authors: Yizhou Dong; Kevin T Love; J Robert Dorkin; Sasilada Sirirungruang; Yunlong Zhang; Delai Chen; Roman L Bogorad; Hao Yin; Yi Chen; Arturo J Vegas; Christopher A Alabi; Gaurav Sahay; Karsten T Olejnik; Weiheng Wang; Avi Schroeder; Abigail K R Lytton-Jean; Daniel J Siegwart; Akin Akinc; Carmen Barnes; Scott A Barros; Mary Carioto; Kevin Fitzgerald; Julia Hettinger; Varun Kumar; Tatiana I Novobrantseva; June Qin; William Querbes; Victor Koteliansky; Robert Langer; Daniel G Anderson
Journal: Proc Natl Acad Sci U S A Date: 2014-02-10 Impact factor: 11.205

4. In vivo endothelial siRNA delivery using polymeric nanoparticles with low molecular weight.

Authors: James E Dahlman; Carmen Barnes; Omar Khan; Aude Thiriot; Siddharth Jhunjunwala; Taylor E Shaw; Yiping Xing; Hendrik B Sager; Gaurav Sahay; Lauren Speciner; Andrew Bader; Roman L Bogorad; Hao Yin; Tim Racie; Yizhou Dong; Shan Jiang; Danielle Seedorf; Apeksha Dave; Kamaljeet S Sandu; Matthew J Webber; Tatiana Novobrantseva; Vera M Ruda; Abigail K R Lytton-Jean; Christopher G Levins; Brian Kalish; Dayna K Mudge; Mario Perez; Ludmila Abezgauz; Partha Dutta; Lynelle Smith; Klaus Charisse; Mark W Kieran; Kevin Fitzgerald; Matthias Nahrendorf; Dganit Danino; Rubin M Tuder; Ulrich H von Andrian; Akin Akinc; Avi Schroeder; Dipak Panigrahy; Victor Kotelianski; Robert Langer; Daniel G Anderson
Journal: Nat Nanotechnol Date: 2014-05-11 Impact factor: 39.213

5. Lipid-like Nanoparticles for Small Interfering RNA Delivery to Endothelial Cells.

Authors: Seung-Woo Cho; Michael Goldberg; Sun Mi Son; Qiaobing Xu; Fan Yang; Ying Mei; Said Bogatyrev; Robert Langer; Daniel G Anderson
Journal: Adv Funct Mater Date: 2009-08-25 Impact factor: 18.808

6. An Orthogonal Array Optimization of Lipid-like Nanoparticles for mRNA Delivery in Vivo.

Authors: Bin Li; Xiao Luo; Binbin Deng; Junfeng Wang; David W McComb; Yimin Shi; Karin M L Gaensler; Xu Tan; Amy L Dunn; Bryce A Kerlin; Yizhou Dong
Journal: Nano Lett Date: 2015-11-06 Impact factor: 11.189

7. Lipid-like nanomaterials for simultaneous gene expression and silencing in vivo.

Authors: Yizhou Dong; Ahmed A Eltoukhy; Christopher A Alabi; Omar F Khan; Omid Veiseh; J Robert Dorkin; Sasilada Sirirungruang; Hao Yin; Benjamin C Tang; Jeisa M Pelet; Delai Chen; Zhen Gu; Yuan Xue; Robert Langer; Daniel G Anderson
Journal: Adv Healthc Mater Date: 2014-03-13 Impact factor: 9.933

8. Therapeutic siRNA silencing in inflammatory monocytes in mice.

Authors: Florian Leuschner; Partha Dutta; Rostic Gorbatov; Tatiana I Novobrantseva; Jessica S Donahoe; Gabriel Courties; Kang Mi Lee; James I Kim; James F Markmann; Brett Marinelli; Peter Panizzi; Won Woo Lee; Yoshiko Iwamoto; Stuart Milstein; Hila Epstein-Barash; William Cantley; Jamie Wong; Virna Cortez-Retamozo; Andita Newton; Kevin Love; Peter Libby; Mikael J Pittet; Filip K Swirski; Victor Koteliansky; Robert Langer; Ralph Weissleder; Daniel G Anderson; Matthias Nahrendorf
Journal: Nat Biotechnol Date: 2011-10-09 Impact factor: 54.908

9. Effects of local structural transformation of lipid-like compounds on delivery of messenger RNA.

Authors: Bin Li; Xiao Luo; Binbin Deng; JoLynn B Giancola; David W McComb; Thomas D Schmittgen; Yizhou Dong
Journal: Sci Rep Date: 2016-02-26 Impact factor: 4.379

10. Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo.

Authors: Hao Yin; Chun-Qing Song; Joseph R Dorkin; Lihua J Zhu; Yingxiang Li; Qiongqiong Wu; Angela Park; Junghoon Yang; Sneha Suresh; Aizhan Bizhanova; Ankit Gupta; Mehmet F Bolukbasi; Stephen Walsh; Roman L Bogorad; Guangping Gao; Zhiping Weng; Yizhou Dong; Victor Koteliansky; Scot A Wolfe; Robert Langer; Wen Xue; Daniel G Anderson
Journal: Nat Biotechnol Date: 2016-02-01 Impact factor: 54.908

5 in total

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

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

2. Report: Optimization study of the preparation factors for argan oil microcapsule based on hybrid-level orthogonal array design via SPSS modeling.

3. Lipopeptide nanoparticles for potent and selective siRNA delivery in rodents and nonhuman primates.

4. In vivo endothelial siRNA delivery using polymeric nanoparticles with low molecular weight.

5. Lipid-like Nanoparticles for Small Interfering RNA Delivery to Endothelial Cells.

6. An Orthogonal Array Optimization of Lipid-like Nanoparticles for mRNA Delivery in Vivo.

7. Lipid-like nanomaterials for simultaneous gene expression and silencing in vivo.

8. Therapeutic siRNA silencing in inflammatory monocytes in mice.

9. Effects of local structural transformation of lipid-like compounds on delivery of messenger RNA.

10. Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo.

Review 1. Nanoscale platforms for messenger RNA delivery.

2. Computational and Experimental Approaches to Investigate Lipid Nanoparticles as Drug and Gene Delivery Systems.

Review 3. Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Therapeutics.

4. Lipid Polymer Hybrid Nanomaterials for mRNA Delivery.

Review 5. mRNA Therapeutic Modalities Design, Formulation and Manufacturing under Pharma 4.0 Principles.