NICKED OR GAPPED NUCLEIC ACID MOLECULES AND USES THEREOF

摘要

The present disclosure provides meroduplex (nicked or gapped) ribonucleic acid molecules (mdRNA) that decreases or silences target gene expression. A n mdRNA of this disclosure comprises at least three strands that combine to form at least two non-overlapping double-stranded regions separated by a nic k or gap wherein one strand is complementary to a target gene RNA. In additi on, the meroduplex may have one or more modifications or substitutions, such as nucleotide base, sugar, terminal cap structure, internucleotide linkage, or any combination of such modifications. Also provided are methods of decr easing expression of a target gene in a cell or in a subject to treat a dise ase related to altered expression of a target gene.