SERPINA1 SIRNAS: COMPOSITIONS OF MATTER AND METHODS OF TREATMENT
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    发明申请
    SERPINA1 SIRNAS: COMPOSITIONS OF MATTER AND METHODS OF TREATMENT 审中-公开
    SERPINA1 SIRNAS:物质组合物和治疗方法

    公开(公告)号:US20140235693A1

    公开(公告)日:2014-08-21

    申请号:US14127782

    申请日:2012-06-22

    IPC分类号: C12N15/113

    摘要: The technology described herein relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the Serpinal gene, and methods of using such dsRNA compositions to inhibit expression of Serpinal. In one embodiment, an iRNA for inhibiting expression of a Serpinal gene includes at least two sequences that are complementary to each other. The iRNA includes a sense strand having a first sequence and an antisense strand having a second sequence. The antisense strand includes a nucleotide sequence that is substantially complementary to at least part of an mRNA encoding Serpinal, and the region of complementarity is 30 nucleotides or less, and at least 15 nucleotides in length. Generally, the iRNA is 19 to 24, e.g., 19 to 21 nucleotides in length.

    摘要翻译: 本文描述的技术涉及靶向Serpinal基因的双链核糖核酸(dsRNA)组合物,以及使用这种dsRNA组合物抑制Serpinal表达的方法。 在一个实施方案中,用于抑制Serpinal基因表达的iRNA包括彼此互补的至少两个序列。 iRNA包括具有第一序列的有义链和具有第二序列的反义链。 反义链包括与编码Serpinal的mRNA的至少一部分基本上互补的核苷酸序列,并且互补区域的长度为30个核苷酸或更少,至少15个核苷酸。 通常,iRNA的长度为19至24,例如19至21个核苷酸。