基因组编辑
清脆的
Cas9
亚基因组mRNA
纳米载体
计算生物学
细胞内
信使核糖核酸
核糖核酸
生物
基因组
细胞生物学
遗传学
基因
药品
药理学
作者
Tianyu Ma,Xianghan Chen,Ming Wang
出处
期刊:ChemBioChem
[Wiley]
日期:2023-03-30
卷期号:24 (9)
被引量:4
标识
DOI:10.1002/cbic.202200801
摘要
Messenger RNA (mRNA) is being used as part of an emerging class of biotherapeutics with great promise for preventing and treating a wide range of diseases, as well as encoding programmable nucleases for genome editing. However, mRNA's low stability and immunogenicity, as well as the impermeability of the cell membrane to mRNA greatly limit mRNA's potential for therapeutic use. Lipid nanoparticles (LNPs) are currently one of the most extensively studied nanocarriers for mRNA delivery and have recently been clinically approved for developing mRNA-based vaccines to prevent COVID-19. In this review, we summarize the latest advances in designing ionizable lipids and formulating LNPs for intracellular and tissue-targeted mRNA delivery. Furthermore, we discuss the progress of intracellular mRNA delivery for spatiotemporally controlled CRISPR/Cas9 genome editing by using LNPs. Finally, we provide a perspective on the future of LNP-based mRNA delivery for CRISPR/Cas9 genome editing and the treatment of genetic disorders.
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