寡核苷酸
核糖核酸酶H
核糖核酸酶P
核糖核酸
生物
基因敲除
RNA干扰
反义RNA
核酸
非编码RNA
长非编码RNA
DNA
计算生物学
分子生物学
遗传学
基因
作者
Rika Maruyama,Toshifumi Yokota
出处
期刊:Methods in molecular biology
日期:2020-01-01
卷期号:: 49-56
被引量:20
标识
DOI:10.1007/978-1-0716-0771-8_3
摘要
Long noncoding RNAs (lncRNAs) are a class of RNA with 200 nucleotides or longer that are not translated into protein. lncRNAs are highly abundant; a study estimates that at least four times more lncRNAs are typically present than coding RNAs in humans. However, function of more than 95% of human lncRNAs are still unknown. Synthetic antisense oligonucleotides called gapmers are powerful tools for lncRNA loss-of-function studies. Gapmers contain a central DNA part, which activates RNase H-mediated RNA degradation, flanked by modified oligonucleotides, such as 2′-O-methyl RNA (2′OMe), 2′-O-methoxyethyl RNA (2′MOE), constrained ethyl nucleosides (cEt), and locked nucleic acids (LNAs). In contrast to siRNA or RNAi-based methods, antisense oligonucleotide gapmer-based knockdown is often more effective against nuclear-localized lncRNA targets, since RNase H is mainly localized in nuclei. As such, gapmers are also potentially a powerful tool for therapeutics targeting lncRNAs in various diseases, including cancer, cardiovascular diseases, lung fibrosis, and neurological/neuromuscular diseases. This chapter will discuss the development and applications of gapmers for lncRNA loss-of-function studies and tips to design effective antisense oligonucleotides.
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