ArticleMolecular therapy. Methods & clinical development2025
Design and systematic evaluation of novel chemical modification patterns enable the identification of a potent siRNA targeting
Article in Molecular therapy. Methods & clinical development, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
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Who cites it
2 citing papers in PubMed.
- Ribofuranose-based GalNAc-conjugated siRNA enhances the liver-targeted delivery and elicits robust RNAi-mediated gene silencing.Molecular therapy. Nucleic acids · 2026Article
- 2'-Ribose-modified nucleotides: A strategy for reducing off-target effects of oligonucleotide drugs.Molecular therapy. Nucleic acids · 2026Article
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8 authors.
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Abstract
Fully modified small interfering RNAs (siRNAs) have demonstrated remarkable advantages in medicine development since they enable the knockdown of the majority of disease-related proteins. However, unmodified siRNAs usually cannot be used for medications due to their biochemical instability. siRNA modification patterns are engineered to enhance both structural stability and pharmacodynamic efficacy, thus being vital in the development of potent siRNA drugs. However, most modification patterns of approved or clinically investigated siRNAs are protected by patents, significantly restricting their broader application. Therefore, the development of proprietary modification patterns has been essential for siRNA therapeutics companies. Here, based on a comprehensive analysis of existing studies, we designed novel modification patterns by altering 2'-F moiety positions at critical sites on sense or antisense strands to enhance siRNA efficacy. Experiments indicate that these modification patterns improve the
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