mRNA Vaccine Era—Mechanisms, Drug Platform and Clinical Prospection DOI Open Access
Shuqin Xu,

Kunpeng Yang,

Rose Li

et al.

International Journal of Molecular Sciences, Journal Year: 2020, Volume and Issue: 21(18), P. 6582 - 6582

Published: Sept. 9, 2020

Messenger ribonucleic acid (mRNA)-based drugs, notably mRNA vaccines, have been widely proven as a promising treatment strategy in immune therapeutics. The extraordinary advantages associated with including their high efficacy, relatively low severity of side effects, and attainment costs, enabled them to become prevalent pre-clinical clinical trials against various infectious diseases cancers. Recent technological advancements alleviated some issues that hinder vaccine development, such efficiency exist both gene translation vivo deliveries. immunogenicity can also be greatly adjusted result upgraded technologies. In this review, we summarized details regarding the optimization underlying biological mechanisms form vaccines. Applications vaccines cancers are introduced. It includes our prospections for applications caused by bacterial pathogens, tuberculosis. At same time, suggestions future development about storage methods, safety concerns, personalized synthesis found context.

Language: Английский

Lipid nanoparticles for mRNA delivery DOI Open Access
Xucheng Hou,

Tal Zaks,

Róbert Langer

et al.

Nature Reviews Materials, Journal Year: 2021, Volume and Issue: 6(12), P. 1078 - 1094

Published: Aug. 10, 2021

Language: Английский

Citations

2234

Delivery technologies for cancer immunotherapy DOI
Rachel Riley, Carl H. June, Róbert Langer

et al.

Nature Reviews Drug Discovery, Journal Year: 2019, Volume and Issue: 18(3), P. 175 - 196

Published: Jan. 8, 2019

Language: Английский

Citations

2134

Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR–Cas gene editing DOI
Qiang Cheng, Tuo Wei, Lukas Farbiak

et al.

Nature Nanotechnology, Journal Year: 2020, Volume and Issue: 15(4), P. 313 - 320

Published: April 1, 2020

Language: Английский

Citations

1492

mRNA vaccines for infectious diseases: principles, delivery and clinical translation DOI Open Access
Namit Chaudhary, Drew Weissman, Kathryn A. Whitehead

et al.

Nature Reviews Drug Discovery, Journal Year: 2021, Volume and Issue: 20(11), P. 817 - 838

Published: Aug. 25, 2021

Language: Английский

Citations

992

Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery DOI Creative Commons
Piotr S. Kowalski, Arnab Rudra, Lei Miao

et al.

Molecular Therapy, Journal Year: 2019, Volume and Issue: 27(4), P. 710 - 728

Published: Feb. 19, 2019

mRNA has broad potential as a therapeutic. Current clinical efforts are focused on vaccination, protein replacement therapies, and treatment of genetic diseases. The translation therapeutics been made possible through advances in the design manufacturing intracellular delivery methods. However, application is still limited by need for improved systems. In this review, we discuss challenges mRNA-based therapeutics, with an emphasis recent biomaterials strategies, present overview applications therapy, gene editing, vaccination.

Language: Английский

Citations

899

Advances in Biomaterials for Drug Delivery DOI
Owen S. Fenton,

Katy N. Olafson,

Padmini S. Pillai

et al.

Advanced Materials, Journal Year: 2018, Volume and Issue: 30(29)

Published: May 7, 2018

Abstract Advances in biomaterials for drug delivery are enabling significant progress biology and medicine. Multidisciplinary collaborations between physical scientists, engineers, biologists, clinicians generate innovative strategies materials to treat a range of diseases. Specifically, recent advances include major breakthroughs cancer immunotherapy, autoimmune diseases, genome editing. Here, the design implementation reviewed. A brief history field is first established, then commentary on RNA delivery, responsive development, immunomodulation provided. Current challenges associated with these areas as well opportunities address long‐standing problems medicine discussed throughout.

Language: Английский

Citations

737

A Novel Amino Lipid Series for mRNA Delivery: Improved Endosomal Escape and Sustained Pharmacology and Safety in Non-human Primates DOI Creative Commons

Staci Sabnis,

E. Sathyajith Kumarasinghe,

Timothy Salerno

et al.

Molecular Therapy, Journal Year: 2018, Volume and Issue: 26(6), P. 1509 - 1519

Published: March 18, 2018

Language: Английский

Citations

618

On the mechanism of tissue-specific mRNA delivery by selective organ targeting nanoparticles DOI Creative Commons
Sean A. Dilliard, Qiang Cheng, Daniel J. Siegwart

et al.

Proceedings of the National Academy of Sciences, Journal Year: 2021, Volume and Issue: 118(52)

Published: Dec. 21, 2021

Significance Liver accumulation represents a significant barrier in the development of therapeutically efficacious nanoparticle drug delivery systems. Using series lipid nanoparticles with distinct organ-targeting properties, we provide evidence for plausible mechanism action to non-liver tissues. Following intravenous injection, specific proteins blood are recruited nanoparticle’s surface based on its molecular composition and they endow it unique biological identity that governs ultimate fate body. An innovative paradigm emerges from this mechanistic understanding delivery—endogenous targeting—wherein is rationally engineered interact overcome liver target organs.

Language: Английский

Citations

565

The clinical progress of mRNA vaccines and immunotherapies DOI Open Access

Ann Barbier,

Allen Yujie Jiang, Peng Zhang

et al.

Nature Biotechnology, Journal Year: 2022, Volume and Issue: 40(6), P. 840 - 854

Published: May 9, 2022

Language: Английский

Citations

492

Systemic nanoparticle delivery of CRISPR-Cas9 ribonucleoproteins for effective tissue specific genome editing DOI Creative Commons
Tuo Wei, Qiang Cheng, Yi-Li Min

et al.

Nature Communications, Journal Year: 2020, Volume and Issue: 11(1)

Published: June 26, 2020

Abstract CRISPR-Cas9 has emerged as a powerful technology that relies on Cas9/sgRNA ribonucleoprotein complexes (RNPs) to target and edit DNA. However, many therapeutic targets cannot currently be accessed due the lack of carriers can deliver RNPs systemically. Here, we report generalizable methodology allows engineering modified lipid nanoparticles efficiently into cells tissues including muscle, brain, liver, lungs. Intravenous injection facilitated tissue-specific, multiplexed editing six genes in mouse High carrier potency was leveraged create organ-specific cancer models livers lungs mice though facile knockout multiple genes. The developed were also able restore dystrophin expression DMD significantly decrease serum PCSK9 level C57BL/6 mice. Application this strategy will facilitate broad nanoparticle development for variety disease amenable protein delivery precise gene correction approaches.

Language: Английский

Citations

474