Recent advances in lipid nanoparticles for delivery of nucleic acid, mRNA, and gene editing-based therapeutics

被引：52

作者：

Mukai, Hidefumi ^{[1
,2
]}

Ogawa, Koki ^{[1
]}

Kato, Naoya ^{[1
]}

Kawakami, Shigeru ^{[1
]}

机构：

[1] Nagasaki Univ, Grad Sch Biomed Sci, Dept Pharmaceut Informat, 1-7-1 Sakamoto, Nagasaki 8528588, Japan

[2] RIKEN Ctr Biosyst Dynam Res, Lab Mol Delivery & Imaging Technol, Chuo Ku, 6-7-3 Minatojima Minamimachi, Kobe, Hyogo 6500047, Japan

来源：

DRUG METABOLISM AND PHARMACOKINETICS | 2022年 / 44卷

关键词：

Lipid nanoparticle; Nucleic acid; mRNA; Gene editing; Base editing; Ionizable lipid; Targeting; DNA barcode; Recombination; Positron emission tomography; IN-VIVO; TARGETED DELIVERY; MANNOSE RECEPTOR; DRUG; DNA; SIRNA; EXPRESSION; SYSTEM; PET; CRISPR;

D O I：

10.1016/j.dmpk.2022.100450

中图分类号：

R9 [药学];

学科分类号：

1007 ;

摘要：

Lipid nanoparticles (LNPs) are becoming popular as a means of delivering therapeutics, including those based on nucleic acids and mRNA. The mRNA-based coronavirus disease 2019 vaccines are perfect ex-amples to highlight the role played by drug delivery systems in advancing human health. The funda-mentals of LNPs for the delivery of nucleic acid-and mRNA-based therapeutics, are well established. Thus, future research on LNPs will focus on addressing the following: expanding the scope of drug de-livery to different constituents of the human body, expanding the number of diseases that can be tar -geted, and studying the change in the pharmacokinetics of LNPs under physiological and pathological conditions. This review article provides an overview of recent advances aimed at expanding the appli-cation of LNPs, focusing on the pharmacokinetics and advantages of LNPs. In addition, analytical tech-niques, library construction and screening, rational design, active targeting, and applicability to gene editing therapy have also been discussed.

引用

页数：13

共 144 条

[1] RNA targeting with CRISPR-Cas13 [J].

Abudayyeh, Omar O. ;

Gootenberg, Jonathan S. ;

Essletzbichler, Patrick ;

Han, Shuo ;

Joung, Julia ;

Belanto, Joseph J. ;

Verdine, Vanessa ;

Cox, David B. T. ;

Kellner, Max J. ;

Regev, Aviv ;

Lander, Eric S. ;

Voytas, Daniel F. ;

Ting, Alice Y. ;

Zhang, Feng .

NATURE, 2017, 550 (7675) :280-+

[2] C2c2 is a single-component programmable RNA-guided RNA-targeting CRISPR effector [J].

Abudayyeh, Omar O. ;

Gootenberg, Jonathan S. ;

Konermann, Silvana ;

Joung, Julia ;

Slaymaker, Ian M. ;

Cox, David B. T. ;

Shmakov, Sergey ;

Makarova, Kira S. ;

Semenova, Ekaterina ;

Minakhin, Leonid ;

Severinov, Konstantin ;

Regev, Aviv ;

Lander, Eric S. ;

Koonin, Eugene V. ;

Zhang, Feng .

SCIENCE, 2016, 353 (6299)

[3] The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs [J].

Akinc, Akin ;

Maier, Martin A. ;

Manoharan, Muthiah ;

Fitzgerald, Kevin ;

Jayaraman, Muthusamy ;

Barros, Scott ;

Ansell, Steven ;

Du, Xinyao ;

Hope, Michael J. ;

Madden, Thomas D. ;

Mui, Barbara L. ;

Semple, Sean C. ;

Tam, Ying K. ;

Ciufolini, Marco ;

Witzigmann, Dominik ;

Kulkarni, Jayesh A. ;

van der Meel, Roy ;

Cullis, Pieter R. .

NATURE NANOTECHNOLOGY, 2019, 14 (12) :1084-1087

[4] Targeted Delivery of RNAi Therapeutics With Endogenous and Exogenous Ligand-Based Mechanisms [J].

Akinc, Akin ;

Querbes, William ;

De, Soma ;

Qin, June ;

Frank-Kamenetsky, Maria ;

Jayaprakash, K. Narayanannair ;

Jayaraman, Muthusamy ;

Rajeev, Kallanthottathil G. ;

Cantley, William L. ;

Dorkin, J. Robert ;

Butler, James S. ;

Qin, LiuLiang ;

Racie, Timothy ;

Sprague, Andrew ;

Fava, Eugenio ;

Zeigerer, Anja ;

Hope, Michael J. ;

Zerial, Marino ;

Sah, Dinah W. Y. ;

Fitzgerald, Kevin ;

Tracy, Mark A. ;

Manoharan, Muthiah ;

Koteliansky, Victor ;

de Fougerolles, Antonin ;

Maier, Martin A. .

MOLECULAR THERAPY, 2010, 18 (07) :1357-1364

[5] A Neutral Envelope-Type Nanoparticle Containing pH-Responsive and SS-Cleavable Lipid-Like Material as a Carrier for Plasmid DNA [J].

Akita, Hidetaka ;

Ishiba, Ryohei ;

Hatakeyama, Hiroto ;

Tanaka, Hiroki ;

Sato, Yusuke ;

Tange, Kota ;

Arai, Masaya ;

Kubo, Kazuhiro ;

Harashima, Hideyoshi .

ADVANCED HEALTHCARE MATERIALS, 2013, 2 (08) :1120-1125

[6] Successful reprogramming of cellular protein production through mRNA delivered by functionalized lipid nanoparticles [J].

Arteta, Marianna Yanez ;

Kjellman, Tomas ;

Bartesaghi, Stefano ;

Wallin, Simonetta ;

Wu, Xiaoqiu ;

Kvist, Alexander J. ;

Dabkowska, Aleksandra ;

Szekely, Noemi ;

Radulescu, Aurel ;

Bergenholtz, Johan ;

Lindfors, Lennart .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2018, 115 (15) :E3351-E3360

[7] Microfluidic Synthesis of Highly Potent Limit-size Lipid Nanoparticles for In Vivo Delivery of siRNA [J].

Belliveau, Nathan M. ;

Huft, Jens ;

Lin, Paulo J. C. ;

Chen, Sam ;

Leung, Alex K. K. ;

Leaver, Timothy J. ;

Wild, Andre W. ;

Lee, Justin B. ;

Taylor, Robert J. ;

Tam, Ying K. ;

Hansen, Carl L. ;

Cullis, Pieter R. .

MOLECULAR THERAPY-NUCLEIC ACIDS, 2012, 1 :e37

[8] Positron emission tomography microdosing:: a new concept with application in tracer and early clinical drug development [J].

Bergström, M ;

Grahnén, A ;

Långström, B .

EUROPEAN JOURNAL OF CLINICAL PHARMACOLOGY, 2003, 59 (5-6) :357-366

[9] ALPHA-4-BETA-7-INTEGRIN MEDIATES LYMPHOCYTE BINDING TO THE MUCOSAL VASCULAR ADDRESSIN MADCAM-1 [J].

BERLIN, C ;

BERG, EL ;

BRISKIN, MJ ;

ANDREW, DP ;

KILSHAW, PJ ;

HOLZMANN, B ;

WEISSMAN, IL ;

HAMANN, A ;

BUTCHER, EC .

CELL, 1993, 74 (01) :185-195

[10] Treatment of influenza and SARS-CoV-2 infections via mRNA-encoded Cas13a in rodents [J].

Blanchard, Emmeline L. ;

Vanover, Daryll ;

Bawage, Swapnil Subhash ;

Tiwari, Pooja Munnilal ;

Rotolo, Laura ;

Beyersdorf, Jared ;

Peck, Hannah E. ;

Bruno, Nicholas C. ;

Hincapie, Robert ;

Michel, Frank ;

Murray, Jackelyn ;

Sadhwani, Heena ;

Vanderheyden, Bob ;

Finn, M. G. ;

Brinton, Margo A. ;

Lafontaine, Eric R. ;

Hogan, Robert J. ;

Zurla, Chiara ;

Santangelo, Philip J. .

NATURE BIOTECHNOLOGY, 2021, 39 (06) :717-726

← 1 2 3 4 5 6 7 8 9 10 →