COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF A GENE FROM THE JC VIRUS
    4.
    发明公开
    COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF A GENE FROM THE JC VIRUS 有权
    抑制JC病毒基因表达的组合物和方法

    公开(公告)号:EP2013222A2

    公开(公告)日:2009-01-14

    申请号:EP07761452.7

    申请日:2007-04-27

    IPC分类号: C07H21/04

    摘要: The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a gene from the JC Virus (JC virus genome), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of a gene from the JC Virus. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by JC virus expression and the expression of a gene from the JC Virus using the pharmaceutical composition; and methods for inhibiting the expression of a gene from the JC Virus in a cell.

    摘要翻译: 本发明涉及用于抑制来自JC病毒(JC病毒基因组)的基因表达的双链核糖核酸(dsRNA),其包含具有长度小于30个核苷酸的核苷酸序列的反义链, 长度为25个核苷酸,并且与来自JC病毒的基因的至少一部分基本互补。 本发明还涉及包含dsRNA和药学上可接受的载体的药物组合物; 使用药物组合物治疗由JC病毒表达引起的疾病和来自JC病毒的基因表达的方法; 以及抑制细胞中来自JC病毒的基因表达的方法。

    COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE PCSK9 GENE
    10.
    发明公开
    COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE PCSK9 GENE 审中-公开
    抑制PCSK9基因表达的组合物和方法

    公开(公告)号:EP2021507A2

    公开(公告)日:2009-02-11

    申请号:EP07762085.4

    申请日:2007-05-14

    摘要: The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a human PCSK9 gene in a cell, wherein said dsRNA comprises at least two sequences that are complementary to each other and wherein a sense strand comprises a first sequence and an antisense strand comprises a second sequence comprising a region of complementarity which is substantially complementary to at least a part of a mRNA encoding PCSK9, and wherein said region of complementarity is less than 30 nucleotides in length and wherein said dsRNA, upon contact with a cell expressing said PCSK9, inhibits expression of said PCSK9 gene, wherein (a) said first sequence is the sequence of SEQ ID NO: 1229 and said second sequence is the sequence of SEQ ID NO: 1230; (b) said first sequence is the sequence of SEQ ID NO: 1227 and said second sequence is the sequence of SEQ ID NO: 1228; or (c) said part of said mRNA encoding PCSK9 is the sequence from position 3530 to 3548 of the mRNA sequence of database entry NM_174936.

    摘要翻译: 本发明涉及用于抑制细胞中人PCSK9基因表达的双链核糖核酸(dsRNA),其中所述dsRNA包含至少两个彼此互补的序列,并且其中有义链包含第一序列和 反义链包含第二序列,所述第二序列包含与编码PCSK9的mRNA的至少一部分基本互补的互补区域,并且其中所述互补区域的长度小于30个核苷酸,并且其中所述dsRNA在与细胞接触时 其中(a)所述第一序列是SEQ ID NO:1229的序列,并且所述第二序列是SEQ ID NO:1230的序列;和(b)表达所述PCSK9抑制所述PCSK9基因的表达。 (b)所述第一序列是SEQ ID NO:1227的序列,并且所述第二序列是SEQ ID NO:1228的序列; 或(c)编码PCSK9的所述mRNA的所述部分是数据库条目NM_174936的mRNA序列的位置3530至3548的序列。