NUCLEIC ACID COMPOUNDS FOR INHIBITING TGFBR GENE EXPRESSION AND USES THEREOF

摘要

The present disclosure provides meroduplex ribonucleic acid molecules (mdRNA) capable of decreasing or silencing TGFBRl, TGFBR2, and/or TGFBR3 gene expression. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-overlapping double-stranded regions separated by a nick or gap wherein one strand is complementary to a TGFBRl, TGFBR2, and/or TGFBR3 mRNA. In addition, the meroduplex may have at least one uridine is a 5-methyluridine, a nucleoside is a locked nucleic acid, or optionally other modifications, and any combination thereof. Also provided are methods of decreasing expression of a TGFBRl, TGFBR2, or TGFBR3 gene in a cell or in a subject to treat a TGFBRl-, TGFBR2-, or TGFBR3 -related disease.