Developing Biodegradable Lipid Nanoparticles for Intracellular mRNA Delivery and Genome Editing,Accounts of Chemical Research

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Developing Biodegradable Lipid Nanoparticles for Intracellular mRNA Delivery and Genome Editing
Accounts of Chemical Research ( IF 16.4 ) Pub Date : 2021-10-20 , DOI: 10.1021/acs.accounts.1c00500
Min Qiu ₁ , Yamin Li ₁ , Hanan Bloomer _{1,

2} , Qiaobing Xu ₁

Affiliation

Since the U.S. Food and Drug Administration (FDA) granted emergency use authorization for two mRNA vaccines against SARS-CoV-2, mRNA-based technology has attracted broad attention from the scientific community to investors. When delivered intracellularly, mRNA has the ability to produce various therapeutic proteins, enabling the treatment of a variety of illnesses, including but not limited to infectious diseases, cancers, and genetic diseases. Accordingly, mRNA holds significant therapeutic potential and provides a promising means to target historically hard-to-treat diseases. Current clinical efforts harnessing mRNA-based technology are focused on vaccination, cancer immunotherapy, protein replacement therapy, and genome editing. The clinical translation of mRNA-based technology has been made possible by leveraging nanoparticle delivery methods. However, the application of mRNA for therapeutic purposes is still challenged by the need for specific, efficient, and safe delivery systems.

中文翻译：

开发用于细胞内 mRNA 传递和基因组编辑的可生物降解脂质纳米颗粒

自美国食品药品监督管理局 (FDA) 授予两种针对 SARS-CoV-2 的 mRNA 疫苗的紧急使用授权以来，基于 mRNA 的技术引起了科学界和投资者的广泛关注。当在细胞内传递时，mRNA 能够产生各种治疗性蛋白质，从而能够治疗多种疾病，包括但不限于传染病、癌症和遗传疾病。因此，mRNA 具有显着的治疗潜力，并为靶向历史上难以治疗的疾病提供了一种有前途的手段。目前利用基于 mRNA 技术的临床工作集中在疫苗接种、癌症免疫疗法、蛋白质替代疗法和基因组编辑上。通过利用纳米颗粒递送方法，基于 mRNA 的技术的临床转化成为可能。

更新日期：2021-11-03

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