核酸
DNA
基因组DNA
引导RNA
分子生物学
信使核糖核酸
突变
化学
清脆的
病毒学
劈理(地质)
生物
遗传学
基因
Cas9
断裂(地质)
古生物学
作者
Dong Yang,Chun‐Qing Song
出处
期刊:Methods in molecular biology
日期:2023-01-01
卷期号:: 159-170
标识
DOI:10.1007/978-1-0716-2879-9_12
摘要
AbstractA genetic disorder is a disease caused by an abnormal DNA sequence, and almost half of the known pathogenic monogenetic mutations are caused by G-to-A mutation (Landrum et al., Nucleic Acids Res 44:D862–868, 2016). Adenine base editors (ABE), developed from the CRISPR, hold the great promise to mediate the A-to-G transition in genomic DNA while not inducing DNA cleavage (Gaudelli et al., Nature 551:464–471, 2017). Additionally, lipid nanoparticles (LNPs), as a non-viral delivery, are able to deliver the ABE mRNAs and gRNA to the target tissues (Newby and Liu, Mol Ther 29:3107–3124, 2021). This chapter mainly introduces the production and LNP delivery of ABE mRNA and gRNA.Key wordsABE mRNALNPsDeliveryMurine liver
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