UNA oligomer structures for therapeutic agents

摘要

This disclosure provides double-stranded RNA complexes having one or more hydroxymethyl substituted nucleomonomer(s) in the passenger strand (or sense strand) of an RNA complex. RNA complexes of the disclosure may be useful for therapeutic applications, diagnostic applications or research applications. RNA complexes include short interfering RNA complexes (siRNA) capable of modulating gene expression comprising an antisense strand and a continuous or a discontinuous passenger strand (“sense strand”). Further, one or more hydroxymethyl substituted nucleomonomer(s) of this disclosure may be positioned at the 3′-end, at the 5′-end, at both the 3′-end and 5′end.

主权项

1. An oligomeric compound comprising one or more acyclic 2′-3′-seco-monomers and one or more ribonucleic acid monomers, the oligomeric compound comprising a sense strand and an antisense strand that together form only one double-stranded region of 15 to 24 base pairs, wherein the 5′-terminal monomer of the sense strand is a terminus of the double-stranded region, wherein any one or more of the two positions at the 5′-end of the sense strand is occupied by the same or different acyclic 2′-3′-seco-monomer, wherein the oligomeric compound has at least one 3′-overhang, and wherein the oligomeric compound is active in RNA interference to modulate expression of a targeted mRNA.