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    PHOSPHOROUS-LINKED OLIGOMERIC COMPOUNDS AND THEIR USE IN GENE MODULATION
    2.
    发明申请
    PHOSPHOROUS-LINKED OLIGOMERIC COMPOUNDS AND THEIR USE IN GENE MODULATION 有权
    磷酸二酯化合物及其在基因调控中的应用

    公开(公告)号：US20160076031A1

    公开(公告)日：2016-03-17

    申请号：US14834873

    申请日：2015-08-25

    申请人： Isis Pharmaceuticals, Inc.

    发明人： Brenda F. Baker ,  Anne B. Eldrup ,  Muthiah Manoharan ,  Balkrishen Bhat ,  Richard H. Griffey ,  Eric E. Swayze ,  Thazha P. Prakash

    IPC分类号： C12N15/113

    CPC分类号： C12N15/111 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/319 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/342 ,  C12N2310/343 ,  C12N2310/346 ,  C12N2310/321 ,  C12N2310/3525 ,  C12N2310/3521

    摘要： Oligonucleotide compositions comprising first and second oligonucleotides are provided wherein at least a portion of the first oligonucleotide is capable of hybridizing with at least a portion of the second oligonucleotide, at least a portion of the first oligonucleotide is complementary to and capable of hybridizing to a selected target nucleic acid, and at least one of the first or second oligonucleotides includes at least one nucleotide having a modified phosphorous-containing internucleoside linkage. Oligonucleotide/protein compositions are also provided comprising an oligonucleotide complementary to and capable of hybridizing to a selected target nucleic acid and at least one protein comprising at least a portion of an RNA-induced silencing complex (RISC), wherein at least one nucleotide of the oligonucleotide has a modified phosphorous-containing internucleoside linkage.

    摘要翻译： 提供了包含第一和第二寡核苷酸的寡核苷酸组合物，其中至少一部分第一寡核苷酸能够与至少一部分第二寡核苷酸杂交，第一寡核苷酸的至少一部分与选定的 靶核酸，并且第一或第二寡核苷酸中的至少一个包含至少一个具有修饰的含磷核苷间键的核苷酸。 还提供了寡核苷酸/蛋白质组合物，其包含与选定的靶核酸互补并且能够与选定的靶核酸杂交的寡核苷酸和至少一种包含至少一部分RNA诱导的沉默复合物（RISC）的蛋白质，其中至少一个核苷酸 寡核苷酸具有修饰的含磷核苷键。

    Modified Short Interfering RNA
    9.
    发明申请
    Modified Short Interfering RNA 有权
    修饰的短干扰RNA

    公开(公告)号：US20090197332A1

    公开(公告)日：2009-08-06

    申请号：US11885782

    申请日：2006-03-08

    申请人： Ioanna Andreou ,  Stefan Pitsch ,  Evelyne Muller ,  Luc Reymond

    发明人： Ioanna Andreou ,  Stefan Pitsch ,  Evelyne Muller ,  Luc Reymond

    IPC分类号： C12N5/06 ,  C07H21/02

    CPC分类号： C12N15/111 ,  C07H19/04 ,  C07H19/10 ,  C07H19/20 ,  C07H21/00 ,  C12N15/113 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2310/318 ,  C12N2310/319 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2320/51 ,  C12Y207/11024 ,  C12N2310/3521 ,  C12N2310/3525

    摘要： The present invention refers to a double-stranded siRNA molecule comprising a sense Strand and an antisense Strand which is essentially complementary to the sense Strand, each of the sense and the antisense Strands comprising at least 17 nucleotides (nt), the siRNA further comprising at least one overhang at the 5′ and/or 3′ end, wherein the overhang residue or overhang residues are chemically modified and selected independently from each other from the group consisting of: (a) 2′-deoxy modified nucleotides; (b) 2′-methoxy modified nucleotides; (c) two nucleosides linked by a 3′ to 5′ or 2′ to 5′ formacetal linkage; (d) nucleotides modified at the 2′-position by a -0-CH2—O—(CH2)2—OH group; and (e) nucleotides comprising in the 3′-position a —CH2—O—(CH2)7—CH3 group.

    摘要翻译： 本发明涉及包含有义链和反义链的双链siRNA分子，其与有义链基本上互补，每个正义和反义链包含至少17个核苷酸（nt），siRNA还包含 在5'和/或3'末端具有至少一个突出端，其中突出端残基或突出端残基被化学修饰并且彼此独立地选自：（a）2'-脱氧修饰的核苷酸; （b）2'-甲氧基修饰的核苷酸; （c）通过3'至5'或2'至5'形式的缩醛键连接的两个核苷; （d）通过-O-CH 2 -O-（CH 2）2 -OH基团在2'-位修饰的核苷酸; 和（e）在3'-位上包含-CH 2 -O-（CH 2）7 -CH 3基团的核苷酸。