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    DELIVERY OF RNAI CONSTRUCTS TO OLIGODENDROCYTES
    4.
    发明申请
    DELIVERY OF RNAI CONSTRUCTS TO OLIGODENDROCYTES 审中-公开
    将RNAI结构递送到寡糖脱乙酰壳多糖

    公开(公告)号：US20110118340A1

    公开(公告)日：2011-05-19

    申请号：US12866444

    申请日：2009-02-06

    申请人： Muthiah Manoharan ,  Kallanthottathil G. Rajeev ,  Dinah Sah ,  William Querbes ,  Pamela Tan ,  Qingmin Chen

    发明人： Muthiah Manoharan ,  Kallanthottathil G. Rajeev ,  Dinah Sah ,  William Querbes ,  Pamela Tan ,  Qingmin Chen

    IPC分类号： A61K31/7088 ,  A61P31/12 ,  A61P25/00 ,  A61P25/18 ,  A61P25/16 ,  A61P25/28

    CPC分类号： C12N15/1131 ,  A61K47/551 ,  A61K47/554 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3515 ,  C12N2320/11 ,  C12N2320/32 ,  C12N2310/3521

    摘要： The invention provides methods for delivering a double-stranded nbonucleic acid (dsRNA) to the central nervous system of a subject, and particularly, to oligodendrocytes of a subject by localized delivery to the brain, e.g., to the corpus caïlosum. For example, the dsRNA molecules can include a first sequence that is selected from the Sroup consisting of the sense sequences of Tables 8, 10, 13-16, and a second sequence selected from the group consisting of the antisense sequences of Tables 8, 10, and 13-16. The dsRNA molecules can include naturally occurring nucleotides or can include at least one modified nucleotide, such as a 2′-O-methyl modified nucleotide, a nucleotide comprising a 5′-phosphorothioate group, or a terminal nucleotide linked to a conjugate group, such as to a cholesteryl derivative or a vitamin E group. Alternatively, the modified nucleotide may be chosen from the group consisting of a 2f-deoxy-2′-fliιioro modified nucleotide, a 2′-de-oxy-modified nucleotide, a locked nucleotide, an abasic nucleotide, 2′-amino-modified nucleotide, 2′-alkyl-modified nucleotide, morpholino nucleotide, a phosphoramidate, and a non-natural bas comprising nucleotide. Generally, such modified sequences will be based on a first sequence of a dsRNA selected from the group consisting of the sense sequences of Tables 8, 10, and 13-16, and a second sequence selected from the group consisting of the antisense sequences of Tables 8 10, and 13-16.

    摘要翻译： 本发明提供了将双链核糖核酸（dsRNA）递送到受试者的中枢神经系统，特别是通过局部递送至脑的例如对胼the体的受试者的少突胶质细胞的方法。 例如，dsRNA分子可以包括选自由表8,10,13-16的有义序列组成的组的第一序列和选自表8,10的反义序列的第二序列 ，和13-16。 dsRNA分子可以包括天然存在的核苷酸，或者可以包括至少一个修饰的核苷酸，例如2'-O-甲基修饰的核苷酸，包含5'-硫代磷酸酯基团的核苷酸或与缀合物连接的末端核苷酸， 关于胆固醇衍生物或维生素E组。 或者，修饰的核苷酸可以选自2f-脱氧-2'-氟修饰的核苷酸，2'-脱氧修饰的核苷酸，锁定的核苷酸，无碱基的核苷酸，2'-氨基修饰的 核苷酸，2'-烷基修饰的核苷酸，吗啉代核苷酸，氨基磷酸酯和包含核苷酸的非天然碱基。 通常，这样的修饰序列将基于选自表8,10和13-16的有义序列的dsRNA的第一序列和选自下表的反义序列的第二序列 8 10和13-16。