| 摘要 |
<p>The present invention relates to a novel siRNA structure and the use thereof, and more particularly to a double-stranded siRNA molecule comprising an antisense strand and a sense strand, wherein the siRNA molecule has at least one single nucleotide bulge formed by introducing a single nucleotide into the antisense strand, particularly at position 2 from the 5' end, and to a method of using the same to silence a target gene. The siRNA molecule of the invention shows high target gene silencing efficiency while minimizing off-target effects caused by the antisense strand, and thus has improved target selectivity. Accordingly, the siRNA molecule of the invention can be substituted for conventional siRNA molecules and can be widely be used in siRNA-based gene silencing techniques, including gene therapy.</p> |
