Journal
ACS MEDICINAL CHEMISTRY LETTERS
Volume 13, Issue 4, Pages 695-700Publisher
AMER CHEMICAL SOC
DOI: 10.1021/acsmedchemlett.2c00027
Keywords
Short interfering RNA; Backbone modification; RNAi; Carrier-free delivery; Phosphate triester
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Funding
- Natural Sciences and Engineering Research Council (NSERC)
- Institute of Rheological Functions of Food in Japan
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siRNAs have shown promise as gene-silencing therapeutics, but their cellular uptake remains a challenge. Researchers synthesized siRNAs with different hydrophobic phosphate triester linkages and evaluated their gene silencing effects without a transfection carrier. The best siRNAs exhibited effective gene silencing.
Short interfering RNAs (siRNAs) show promise as gene-silencing therapeutics, but their cellular uptake remains achallenge. We have recently shown the synthesis of siRNAs bearing a single neutral phenylethyl phosphotriester linkage within thesense strand. Here, we report the synthesis of siRNAs bearing three different hydrophobic phosphate triester linkages at keypositions within the sense strand and assess their gene silencing in the absence of a transfection carrier. The best siRNAs bearinghydrophobic phosphate triester tails were not aromatic and exhibited effective gene silencing (IC50 approximate to 56-141 nM), whereas thearomatic derivative with three hydrophobic tails did not exhibit carrier-free gene silencing
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