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    COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF A GENE FROM THE JC VIRUS
    3.
    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

    申请人： Alnylam Pharmaceuticals Inc.

    发明人： TAN, Pamela ,  SAH, Dinah ,  BRAMLAGE, Birgit

    IPC分类号： C07H21/04

    CPC分类号： C12N15/1131 ,  A61K48/005 ,  C07H21/02 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3521

    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
    9.
    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

    申请人： Alnylam Pharmaceuticals Inc.

    发明人： TAN, Pamela ,  BRAMLAGE, Birgit ,  FRANK-KAMENETSKY, Maria ,  FITZGERALD, Kevin ,  AKINC, Akin ,  KOTELIANSKI, Victor, E.

    IPC分类号： C12Q1/68 ,  A01N43/04 ,  C07H21/04 ,  A61K31/07

    CPC分类号： C12N15/1137 ,  A61K31/713 ,  C12N2310/111 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/332 ,  C12N2310/3515 ,  C12N2310/3521

    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。