OLIGONUCLEOTIDE COMPOUNDS COMPRISING NON-NUCLEOTIDE OVERHANGS

摘要

The invention relates to siRNA compounds comprising one non-nucleotide moiety covalently attached to at least one of the sense or antisense strands to down-regulate the expression of human genes. The invention also relates to pharmaceutical compositions comprising such compounds and a pharmaceutically acceptable carrier and to methods of treating and/or preventing the incidence or severity of various diseases or conditions associated with the target genes and/or symptoms associated with such diseases or conditions.

主权项

1. A double-stranded nucleic acid molecule comprising a sense strand sequence 5′ CAGAAGUCAUCUUGCUACA 3′ (SEQ ID NO. 9) and an antisense strand sequence 5′ UGUAGCAAGAUGACUUCUG 3′ (SEQ ID NO. 10); or a pharmaceutically acceptable salt of such molecule.