化学
基因沉默
基因敲除
小干扰RNA
寡核苷酸
N6-甲基腺苷
RNA干扰
甲基化
核糖核酸
组合化学
生物化学
基因
甲基转移酶
作者
Anna M. Rydzik,Doris Riether,Dirk Gottschling
标识
DOI:10.1016/j.bmcl.2023.129126
摘要
A synthesis of 2'-fluoro and 2'-methoxy N6-methyladenosine phosphoramidites and their successful incorporation into oligonucleotides is reported. 2'-fluoro and 2́-methoxy modifications of sugars in siRNAs are known to aid stability and N6-methylation modifies the potency of therapeutic silencing RNAs (siRNA). We demonstrate that a combination of those modifications incorporated into the antisense strand of siRNA leads to efficient knockdown of a target gene in cells. This work broadens the available pool of chemical modifications of therapeutic siRNAs and provides tools for their efficient synthesis.
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