Gene editing therapeutics based on mRNA delivery

Popovitz, Juliana; Sharma, Rohit; Hoshyar, Reyhane; Kim, Beob Soo; Murthy, Niren; Lee, Kunwoo

doi:10.1016/j.addr.2023.115026

Cited by 15 publications

(5 citation statements)

References 103 publications

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“…Common factors include delivery efficiency, stability, dose optimization, and biological safety. [ 119–121 ] However, there are specific requirements for in vitro and in vivo delivery: [ 122–124 ] 1) Delivery targets: In vitro delivery typically occurs within cells, while in vivo therapy requires precise delivery of mRNA formulations to specific targets within the body. 2) Biological distribution: Consideration must be given to the distribution and metabolism of carrier‐mediated mRNA drugs within the biological system.…”

Section: Construction Of Engineered Mrna Delivery Systemsmentioning

confidence: 99%

Engineered mRNA Delivery Systems for Biomedical Applications

Wang,

Zhu,

Gan

et al. 2024

Advanced Materials

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Messenger RNA (mRNA)‐based therapeutic strategies have shown remarkable promise in preventing and treating a staggering range of diseases. Optimizing the structure and delivery system of engineered mRNA has greatly improved its stability, immunogenicity, and protein expression levels, which has led to a wider range of uses for mRNA therapeutics. In this tutorial review, we first provide a thorough analysis of the optimization strategies used in the structure of mRNA and then describe delivery systems in great detail. Furthermore, we summarize the latest advancements in biomedical engineering for mRNA technology, including its applications in combatting infectious diseases, treating cancer, providing protein replacement therapy, conducting gene editing, and more. Lastly, we offer a perspective on forthcoming challenges and prospects concerning the advancement of mRNA therapeutics. Despite these challenges, mRNA‐based therapeutics remain promising, with the potential to revolutionize disease treatment and contribute to significant advancements in the biomedical field.This article is protected by copyright. All rights reserved

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Section: Construction Of Engineered Mrna Delivery Systemsmentioning

confidence: 99%

Engineered mRNA Delivery Systems for Biomedical Applications

Wang,

Zhu,

Gan

et al. 2024

Advanced Materials

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“…RNA-based delivery of genome-editing components has several promising features, including smaller molecular structures, rapid onset, and mitigation of off-target effects that are caused by persistent expression of Cas proteins. 294 , 295 The delivery of genome editors in the form of mRNA is currently a widely pursued strategy. Delivering of mRNA encoding the genome-editing reagents can be achieved via various approaches, such as microinjection or electroporation, and by using non-viral vectors such as lipid nanoparticles (LNP) and newly developed virus like particles (VLPs) (Fig.…”

Section: Delivery Of Precise Genome-editing Reagentsmentioning

confidence: 99%

Precise genome-editing in human diseases: mechanisms, strategies and applications

Zheng,

Li,

Zhou

et al. 2024

Sig Transduct Target Ther

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Precise genome-editing platforms are versatile tools for generating specific, site-directed DNA insertions, deletions, and substitutions. The continuous enhancement of these tools has led to a revolution in the life sciences, which promises to deliver novel therapies for genetic disease. Precise genome-editing can be traced back to the 1950s with the discovery of DNA’s double-helix and, after 70 years of development, has evolved from crude in vitro applications to a wide range of sophisticated capabilities, including in vivo applications. Nonetheless, precise genome-editing faces constraints such as modest efficiency, delivery challenges, and off-target effects. In this review, we explore precise genome-editing, with a focus on introduction of the landmark events in its history, various platforms, delivery systems, and applications. First, we discuss the landmark events in the history of precise genome-editing. Second, we describe the current state of precise genome-editing strategies and explain how these techniques offer unprecedented precision and versatility for modifying the human genome. Third, we introduce the current delivery systems used to deploy precise genome-editing components through DNA, RNA, and RNPs. Finally, we summarize the current applications of precise genome-editing in labeling endogenous genes, screening genetic variants, molecular recording, generating disease models, and gene therapy, including ex vivo therapy and in vivo therapy, and discuss potential future advances.

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“…This technique relies on genetically engineered nucleases, which generate site-specific double-strand breaks at specific locations in the genome, inducing the organism to repair these breaks through non-homologous end joining or homologous recombination. 96–98…”

Section: The Application Of Micro/nano-electroporation In Gene Therapymentioning

confidence: 99%