Intracellular Routing and Recognition of Lipid-Based mRNA Nanoparticles

被引：41

作者：

Delehedde, Christophe ^{[1
,2
]}

Even, Luc ^{[2
]}

Midoux, Patrick ^{[1
]}

Pichon, Chantal ^{[1
]}

Perche, Federico ^{[1
]}

机构：

[1] CNRS, Ctr Biophys Mol, Innovat Therapies & Nanomed, UPR4301, Rue Charles Sadron, F-45071 Orleans, France

[2] Sanofi R&D, Integrated Drug Discovery, F-91385 Chilly Mazarin, France

来源：

PHARMACEUTICS | 2021年 / 13卷 / 07期

关键词：

mRNA delivery; intracellular routing; lipid-based nanoparticles; MEDIATED SIRNA DELIVERY; GENE DELIVERY; IN-VITRO; ENDOSOMAL ESCAPE; ENDOCYTOSIS; TRAFFICKING; EXPRESSION; CELLS; THERAPEUTICS; VIVO;

D O I：

10.3390/pharmaceutics13070945

中图分类号：

R9 [药学];

学科分类号：

1007 ;

摘要：

Messenger RNA (mRNA) is being extensively used in gene therapy and vaccination due to its safety over DNA, in the following ways: its lack of integration risk, cytoplasmic expression, and transient expression compatible with fine regulations. However, clinical applications of mRNA are limited by its fast degradation by nucleases, and the activation of detrimental immune responses. Advances in mRNA applications, with the recent approval of COVID-19 vaccines, were fueled by optimization of the mRNA sequence and the development of mRNA delivery systems. Although delivery systems and mRNA sequence optimization have been abundantly reviewed, understanding of the intracellular processing of mRNA is mandatory to improve its applications. We will focus on lipid nanoparticles (LNPs) as they are the most advanced nanocarriers for the delivery of mRNA. Here, we will review how mRNA therapeutic potency can be affected by its interactions with cellular proteins and intracellular distribution.

引用

页数：26

共 141 条

[1] 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

[2] Development of an SS-Cleavable pH-Activated Lipid-Like Material (ssPalm) as a Nucleic Acid Delivery Device [J].

Akita, Hidetaka .

BIOLOGICAL & PHARMACEUTICAL BULLETIN, 2020, 43 (11) :1617-1625

[3] Spatial and Temporal Control of CRISPR-Cas9-Mediated Gene Editing Delivered via a Light-Triggered Liposome System [J].

Aksoy, Yagiz Alp ;

Yang, Biyao ;

Chen, Wenjie ;

Hung, Tzongtyng ;

Kuchel, Rhiannon P. ;

Zammit, Nathan W. ;

Grey, Shane T. ;

Goldys, Ewa M. ;

Deng, Wei .

ACS APPLIED MATERIALS & INTERFACES, 2020, 12 (47) :52433-52444

[4] Nucleoside modifications in RNA limit activation of 2'-5'-oligoadenylate synthetase and increase resistance to cleavage by RNase L [J].

Anderson, Bart R. ;

Muramatsu, Hiromi ;

Jha, Babal K. ;

Silverman, Robert H. ;

Weissman, Drew ;

Kariko, Katalin .

NUCLEIC ACIDS RESEARCH, 2011, 39 (21) :9329-9338

[5] Designing biomaterials for the delivery of RNA therapeutics to stimulate bone healing [J].

Andree, L. ;

Yang, F. ;

Brock, R. ;

Leeuwenburgh, S. C. G. .

MATERIALS TODAY BIO, 2021, 10 (10)

[6] Light-triggered switching of liposome surface charge directs delivery of membrane impermeable payloads in vivo [J].

Arias-Alpizar, Gabriela ;

Kong, Li ;

Vlieg, Redmar C. ;

Rabe, Alexander ;

Papadopoulou, Panagiota ;

Meijer, Michael S. ;

Bonnet, Sylvestre ;

Vogel, Stefan ;

van Noort, John ;

Kros, Alexander ;

Campbell, Frederick .

NATURE COMMUNICATIONS, 2020, 11 (01)

[7] Expression of a mannose/fucose membrane lectin on human dendritic cells [J].

Avrameas, A ;

McIlroy, D ;

Hosmalin, A ;

Autran, B ;

Debre, P ;

Monsigny, M ;

Roche, AC ;

Midoux, P .

EUROPEAN JOURNAL OF IMMUNOLOGY, 1996, 26 (02) :394-400

[8] Synthesis of a trimannosylated-equipped archaeal diether lipid for the development of novel glycoliposomes [J].

Barbeau, Julie ;

Lemiegre, Lok ;

Quelen, Allan ;

Malard, Virginie ;

Gao, Haifei ;

Goncalves, Cristine ;

Berchel, Mathieu ;

Jaffres, Paul-Alain ;

Pichon, Chantal ;

Midoux, Patrick ;

Benvegnu, Thierry .

CARBOHYDRATE RESEARCH, 2016, 435 :142-148

[9] Encapsulation state of messenger RNA inside lipid nanoparticles [J].

Brader, Mark L. ;

Williams, Sean J. ;

Banks, Jessica M. ;

Hui, Wong H. ;

Zhou, Z. Hong ;

Jin, Lin .

BIOPHYSICAL JOURNAL, 2021, 120 (14) :2766-2770

[10] Nanomaterial Delivery Systems for mRNA Vaccines [J].

Buschmann, Michael D. ;

Carrasco, Manuel J. ;

Alishetty, Suman ;

Paige, Mikell ;

Alameh, Mohamad Gabriel ;

Weissman, Drew .

VACCINES, 2021, 9 (01) :1-30

← 1 2 3 4 5 6 7 8 9 10 →