基因沉默
小干扰RNA
反式siRNA
RNA干扰
化学
感应(电子)
感觉链
转染
基因
细胞生物学
生物物理学
核糖核酸
生物化学
生物
物理化学
作者
Matthew L. Hammill,Kouta Tsubaki,Lidya Salim,Andrew Varley,Ifrodet Giorgees,Masaya Kitamura,Tatsuo Okauchi,Jean‐Paul Desaulniers
标识
DOI:10.1021/acsmedchemlett.2c00027
摘要
Short interfering RNAs (siRNAs) show promise as gene-silencing therapeutics, but their cellular uptake remains a challenge. We have recently shown the synthesis of siRNAs bearing a single neutral phenylethyl phosphotriester linkage within the sense strand. Here, we report the synthesis of siRNAs bearing three different hydrophobic phosphate triester linkages at key positions within the sense strand and assess their gene silencing in the absence of a transfection carrier. The best siRNAs bearing hydrophobic phosphate triester tails were not aromatic and exhibited effective gene silencing (IC50 ≈ 56-141 nM), whereas the aromatic derivative with three hydrophobic tails did not exhibit carrier-free gene silencing.
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