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    Modified triple-helix forming oligonucleotides for targeted mutagenesis
    1.
    发明授权
    Modified triple-helix forming oligonucleotides for targeted mutagenesis 有权
    用于靶向诱变的修饰的三螺旋形成寡核苷酸

    公开(公告)号：US08658608B2

    公开(公告)日：2014-02-25

    申请号：US11562849

    申请日：2006-11-22

    申请人： Peter M. Glazer ,  Michael M. Siedman

    发明人： Peter M. Glazer ,  Michael M. Siedman

    IPC分类号： C12N15/11 ,  C07H21/04 ,  C12N5/00

    CPC分类号： C12N15/102 ,  C12N15/111 ,  C12N15/113 ,  C12N2310/152 ,  C12N2310/321 ,  C12N2310/33 ,  C12N2320/12 ,  C12N2320/13

    摘要： High affinity, chemically modified triplex-forming oligonucleotides (TFOs) and methods for use thereof are disclosed. TFOs are defined as triplex-forming oligonucleotides which bind as third strands to duplex DNA in a sequence specific manner. Triplex-forming oligonucleotides may be comprised of any possible combination of nucleotides and modified nucleotides. Modified nucleotides may contain chemical modifications of the heterocyclic base, sugar moiety or phosphate moiety. A high affinity oligonucleotide (Kd≦2×10−8) which forms a triple strand with a specific DNA segment of a target gene DNA is generated. It is preferable that the Kd for the high affinity oligonucleotide is below 2×10−10. The nucleotide binds or hybridizes to a target sequence within a target gene or target region of a chromosome, forming a triplex region. The binding of the oligonucleotide to the target region stimulates mutations within or adjacent to the target region using cellular DNA synthesis, recombination, and repair mechanisms. The mutation generated activates, inactivates, or alters the activity and function of the target gene.

    摘要翻译： 公开了高亲和力，化学修饰的三链体形成寡核苷酸（TFO）及其使用方法。 TFO被定义为以序列特异性方式结合为第二链的双链DNA的三重复合形成寡核苷酸。 三重复合形成寡核苷酸可以由核苷酸和经修饰的核苷酸的任何可能组合组成。 修饰的核苷酸可以包含杂环碱基，糖部分或磷酸酯部分的化学修饰。 产生与目标基因DNA的特定DNA区段形成三链的高亲和力寡核苷酸（Kd @ 2×10-8）。 高亲和力寡核苷酸的Kd优选低于2×10-10。 该核苷酸与染色体的靶基因或靶区域中的靶序列结合或杂交，形成三重区域。 寡核苷酸与靶区域的结合使用细胞DNA合成，重组和修复机制刺激靶区域内或邻近靶区域的突变。 产生的突变会激活，失活或改变靶基因的活性和功能。

    COMPOSITIONS AND METHODS FOR INHIBITING THE EXPRESSION OF ANTI-APOPTOPIC GENES
    3.
    发明申请
    COMPOSITIONS AND METHODS FOR INHIBITING THE EXPRESSION OF ANTI-APOPTOPIC GENES 有权
    用于抑制抗病毒基因表达的组合物和方法

    公开(公告)号：US20090053808A1

    公开(公告)日：2009-02-26

    申请号：US12175938

    申请日：2008-07-18

    申请人： Hans-Peter Vornlocher

    发明人： Hans-Peter Vornlocher

    IPC分类号： A61K31/7105 ,  C12N5/06 ,  A61P31/00

    CPC分类号： C12N15/113 ,  C12N15/1131 ,  C12N2310/152 ,  C12N2310/315 ,  C12N2310/3183 ,  C12N2310/33 ,  C12N2310/351 ,  C12N2310/3511 ,  C12N2310/53

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

    摘要翻译： 本发明涉及用于抑制抗凋亡基因表达的双链核糖核酸（dsRNA），其包含具有长度小于25个核苷酸的核苷酸序列的反义链，其与至少一个 凋亡基因的一部分，如Bcl基因。 本发明还涉及包含dsRNA与药学上可接受的载体的药物组合物; 用于使用该药物组合物治疗由抗凋亡基因表达引起的疾病的方法; 以及抑制细胞中抗凋亡基因表达的方法。

    MODIFIED TRIPLE-HELIX FORMING OLIGONUCLEOTIDES FOR TARGETED MUTAGENESIS
    4.
    发明申请
    MODIFIED TRIPLE-HELIX FORMING OLIGONUCLEOTIDES FOR TARGETED MUTAGENESIS 有权
    修饰三肽螺旋形成针对性突变体的寡核苷酸

    公开(公告)号：US20070219122A1

    公开(公告)日：2007-09-20

    申请号：US11562849

    申请日：2006-11-22

    申请人： Peter Glazer ,  Michael Siedman

    发明人： Peter Glazer ,  Michael Siedman

    IPC分类号： A61K48/00 ,  C12N15/09 ,  C12N5/10

    CPC分类号： C12N15/102 ,  C12N15/111 ,  C12N15/113 ,  C12N2310/152 ,  C12N2310/321 ,  C12N2310/33 ,  C12N2320/12 ,  C12N2320/13

    摘要： High affinity, chemically modified triplex-forming oligonucleotides (TFOs) and methods for use thereof are disclosed. TFOs are defined as triplex-forming oligonucleotides which bind as third strands to duplex DNA in a sequence specific manner. Triplex-forming oligonucleotides may be comprised of any possible combination of nucleotides and modified nucleotides. Modified nucleotides may contain chemical modifications of the heterocyclic base, sugar moiety or phosphate moiety. A high affinity oligonucleotide (Kd≦2×10−8) which forms a triple strand with a specific DNA segment of a target gene DNA is generated. It is preferable that the Kd for the high affinity oligonucleotide is below 2×10−10. The nucleotide binds or hybridizes to a target sequence within a target gene or target region of a chromosome, forming a triplex region. The binding of the oligonucleotide to the target region stimulates mutations within or adjacent to the target region using cellular DNA synthesis, recombination, and repair mechanisms. The mutation generated activates, inactivates, or alters the activity and function of the target gene.

    摘要翻译： 公开了高亲和力，化学修饰的三链体形成寡核苷酸（TFO）及其使用方法。 TFO被定义为以序列特异性方式结合为第二链的双链DNA的三重复合形成寡核苷酸。 三重复合形成寡核苷酸可以由核苷酸和经修饰的核苷酸的任何可能组合组成。 修饰的核苷酸可以包含杂环碱基，糖部分或磷酸酯部分的化学修饰。 产生与目标基因DNA的特异性DNA区段形成三链的高亲和性寡核苷酸（K 2×2×10 -8 -8）。 优选的是，高亲和力寡核苷酸的K 低于2×10 -10。 该核苷酸与染色体的靶基因或靶区域中的靶序列结合或杂交，形成三重区域。 寡核苷酸与靶区域的结合使用细胞DNA合成，重组和修复机制刺激靶区域内或邻近靶区域的突变。 产生的突变会激活，失活或改变靶基因的活性和功能。

    TREATMENT OF PARKINSON'S DISEASE WITH OLIGONUCLEOTIDES
    7.
    发明申请
    TREATMENT OF PARKINSON'S DISEASE WITH OLIGONUCLEOTIDES 审中-公开
    用寡核苷酸治疗帕金森病

    公开(公告)号：US20030060436A1

    公开(公告)日：2003-03-27

    申请号：US09435249

    申请日：1999-11-05

    申请人： CLIFFORD KENT WEBER ESQ.

    发明人： JAY S. SCHNEIDER

    IPC分类号： A61K048/00

    CPC分类号： A61K31/711 ,  A61K38/00 ,  C12N15/1137 ,  C12N2310/152 ,  C12N2310/315

    摘要： The present invention relates to a method of treatment of Parkinson's disease, and to the use of antisense oligonucleotides or triplex oligonucleotides introduced into targeted brain structures to decrease the function of brain circuits known to be overactive in the Parkinsonian brain. Antisense or triplex oligonucleotides are targeted to the internal globus pallidus and/or substantia nigra pars reticulata (SNr) where the expression of glutamic acid decarboxylase (GAD67, GAD65, or a combination of the two isoforms) is downregulated. The present invention also relates to a method of treatment of Parkinson's disease where antisense or triplex oligonucleotides are targeted to the internal globus pallidus and/or substantia nigra pars reticulata for the downregulation of glutamate receptors. The present invention further relates to a method of treatment of Parkinson's disease where antisense or triplex oligonucleotides are targeted to the thatlamic motor nuclei for the downregulation of GABA receptors.

    摘要翻译： 本发明涉及治疗帕金森病的方法，以及引入靶向脑结构中的反义寡核苷酸或三链寡核苷酸的用途，以降低已知在帕金森病脑中过度活跃的脑电路的功能。 反义或三重寡核苷酸靶向内部苍白球和/或黑质浆果（SNr），其中谷氨酸脱羧酶（GAD67，GAD65或两种同种型的组合）的表达下调。 本发明还涉及一种治疗帕金森病的方法，其中反义或三重寡核苷酸针对内部苍白球和/或黑质黑质对谷氨酸受体的下调。 本发明还涉及治疗帕金森病的方法，其中反义或三重寡核苷酸靶向用于下调GABA受体的该运动细胞核。

    Compositions and methods for inhibiting expression of anti-apoptotic genes
    10.
    发明授权
    Compositions and methods for inhibiting expression of anti-apoptotic genes 有权
    抑制抗凋亡基因表达的组合物和方法

    公开(公告)号：US08143390B2

    公开(公告)日：2012-03-27

    申请号：US12961337

    申请日：2010-12-06

    申请人： Hans-Peter Vornlocher

    发明人： Hans-Peter Vornlocher

    IPC分类号： C07H21/04 ,  C07H21/02 ,  C12N15/63 ,  C12N15/85

    CPC分类号： C12N15/113 ,  C12N15/1131 ,  C12N2310/152 ,  C12N2310/315 ,  C12N2310/3183 ,  C12N2310/33 ,  C12N2310/351 ,  C12N2310/3511 ,  C12N2310/53

    摘要： The present invention relates to an isolated double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of bcl-2, where the antisense strand comprises a sequence that comprises a region of complementarity which is substantially complementary to at least a part of an mRNA encoding bcl-2. The dsRNA, upon contact with a cell expressing the bcl-2, inhibits expression of the bcl-2 gene by at least 20%. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier. The invention further relates to a vector for inhibiting expression of bcl-2 in a cell, where the vector comprises a regulatory sequence operably linked to a nucleotide sequence that encodes at least one strand of the dsRNA.

    摘要翻译： 本发明涉及用于抑制bcl-2表达的分离的双链核糖核酸（dsRNA），其中反义链包含一个序列，该序列包含与至少一部分mRNA编码基本互补的互补区 bcl-2。 当与表达bcl-2的细胞接触时，dsRNA抑制bcl-2基因的表达至少20％。 本发明还涉及包含dsRNA与药学上可接受的载体的药物组合物。 本发明还涉及用于抑制细胞中bcl-2表达的载体，其中载体包含与编码dsRNA的至少一条链的核苷酸序列可操作地连接的调节序列。