Synthesis and Properties of RNA Modified with Cationic Amine Internucleoside Linkage.

ABSTRACT: Chemical modifications of RNA are important tools for the development of RNA therapeutics. The present study reports a novel RNA backbone modification that replaces the negatively charged phosphate with a positively charged amine linkage. Despite being thermally destabilizing in RNA duplexes, the amine linkage caused a relatively modest decrease of activity of a modified short interfering RNA (siRNA). At position 2 of the guide strand, the amine modification strongly enhanced the specificity of siRNA while causing an ∼5-fold drop of on-target activity. These results support the future development of amines as cationic RNA modifications and novel tools to modulate protein-RNA interactions.

SUBMITTER: Pal C 

PROVIDER: S-EPMC11058646 | biostudies-literature | 2024 Feb

REPOSITORIES: biostudies-literature