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    Compositions and Methods For Inhibiting Expression Of A Target Gene
    32.
    发明申请
    Compositions and Methods For Inhibiting Expression Of A Target Gene 有权
    抑制靶基因表达的组成和方法

    公开(公告)号：US20110111493A1

    公开(公告)日：2011-05-12

    申请号：US12894018

    申请日：2010-09-29

    申请人： Roland Kreutzer ,  Stefan Limmer ,  Sylvia Limmer ,  Philipp Hadwiger

    发明人： Roland Kreutzer ,  Stefan Limmer ,  Sylvia Limmer ,  Philipp Hadwiger

    IPC分类号： C12N5/09 ,  C07H21/02

    CPC分类号： C12N15/1138 ,  A61K38/00 ,  C12N15/111 ,  C12N15/1131 ,  C12N2310/14 ,  C12N2310/53 ,  C12N2320/50

    摘要： The present invention relates to a double-stranded ribonucleic acid (dsRNA) having a nucleotide sequence which is substantially identical to at least a part of a target gene and which is no more than 49, preferably less than 25, nucleotides in length, and which comprises a complementary (antisense) RNA strand having a 1 to 4 nucleotide overhang at the 3′-end and a blunt 5′-end. The invention further relates to a pharmaceutical composition comprising the dsRNA and a pharmaceutically acceptable carrier. The pharmaceutical compositions are useful for inhibiting the expression of a target gene, as well as for treating diseases caused by expression of the target gene, at low dosages (i.e., less than 5 milligrams, preferably less than 25 micrograms, per kg body weight per day). The invention also relates to methods for inhibiting the expression of a target gene, as well as methods for treating diseases caused by the expression of the gene.

    摘要翻译： 本发明涉及具有与靶基因的至少一部分基本上相同且长度不超过49个，优选小于25个核苷酸的核苷酸序列的双链核糖核酸（dsRNA），其中 包含在3'末端具有1至4个核苷酸突出端和5'-末端的互补（反义）RNA链。 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。 药物组合物可用于抑制靶基因的表达，以及用于治疗由靶基因表达引起的疾病，以低剂量（即，小于5毫克，优选小于25微克，每kg体重/ 天）。 本发明还涉及抑制靶基因表达的方法，以及用于治疗由基因表达引起的疾病的方法。

    DOUBLE-STRANDED RNA (dsRNA) AND METHOD OF USE FOR INHIBITING EXPRESSION OF A FUSION GENE
    33.
    发明申请
    DOUBLE-STRANDED RNA (dsRNA) AND METHOD OF USE FOR INHIBITING EXPRESSION OF A FUSION GENE 审中-公开
    双链RNA（dsRNA）和用于抑制融合基因表达的方法

    公开(公告)号：US20110065777A1

    公开(公告)日：2011-03-17

    申请号：US12912616

    申请日：2010-10-26

    申请人： Olaf Heidenreich ,  Hans-Peter Vornlocher ,  Roland Kreutzer ,  Stefan Limmer

    发明人： Olaf Heidenreich ,  Hans-Peter Vornlocher ,  Roland Kreutzer ,  Stefan Limmer

    IPC分类号： A61K31/713 ,  C07H21/02 ,  A61P35/00 ,  C12N5/071 ,  C12N5/078

    CPC分类号： C12N15/1135 ,  A61K38/00 ,  C12N2310/14 ,  C12N2310/3183 ,  C12N2310/53

    摘要： Specific inhibition of expression of a fusion gene in mammals occurs using a short, double-stranded ribonucleic acid molecule (dsRNA). The dsRNA comprises two separate non-linked RNA strands, an 51 strand and a complementary strand. The strands are 20 to 23 nucleotides in length, and the 51 strand is complementary to the fusion junction of the AML-1/MTG-8 fusion gene. The dsRNA also comprises at least 3 nucleotides on each side of the fusion junction. The dsRNA can be introduced into and maintained in a mammalian cell under conditions and for a time sufficient to obtain degradation of mRNA of the fusion gene to inhibit expression of the fusion gene. The dsRNAs and methods described are useful for treating diseases caused by chromosomal aberrations, particularly malignant diseases such as lymphoma and leukemia.

    摘要翻译： 使用短的双链核糖核酸分子（dsRNA）发生融合基因在哺乳动物中的表达的特异性抑制。 dsRNA包含两个单独的非连接的RNA链，51链和互补链。 链长为20至23个核苷酸，51链与AML-1 / MTG-8融合基因的融合连接互补。 dsRNA还在融合连接的每一侧上包含至少3个核苷酸。 可以在足以获得融合基因mRNA降解以抑制融合基因表达的条件下和时间内将dsRNA引入哺乳动物细胞并保持在哺乳动物细胞中。 描述的dsRNA和方法可用于治疗由染色体畸变引起的疾病，特别是恶性疾病如淋巴瘤和白血病。

    Compositions and methods for inhibiting expression of a target gene
    37.
    发明授权
    Compositions and methods for inhibiting expression of a target gene 有权
    用于抑制靶基因表达的组合物和方法

    公开(公告)号：US07829693B2

    公开(公告)日：2010-11-09

    申请号：US10384339

    申请日：2003-03-07

    申请人： Roland Kreutzer ,  Stefan Limmer ,  Sylvia Limmer ,  Philipp Hadwiger

    发明人： Roland Kreutzer ,  Stefan Limmer ,  Sylvia Limmer ,  Philipp Hadwiger

    IPC分类号： C12Q1/68 ,  C12N15/88 ,  C07H21/02 ,  C07H21/04

    CPC分类号： C12N15/1138 ,  A61K38/00 ,  C12N15/111 ,  C12N15/1131 ,  C12N2310/14 ,  C12N2310/53 ,  C12N2320/50

    摘要： The present invention relates to a double-stranded ribonucleic acid (dsRNA) having a nucleotide sequence which is substantially identical to at least a part of a target gene and which is no more than 49, preferably less than 25, nucleotides in length, and which comprises a complementary (antisense) RNA strand having a 1 to 4 nucleotide overhang at the 3′-end and a blunt 5′-end. The invention further relates to a pharmaceutical composition comprising the dsRNA and a pharmaceutically acceptable carrier. The pharmaceutical compositions are useful for inhibiting the expression of a target gene, as well as for treating diseases caused by expression of the target gene, at low dosages (i.e., less than 5 milligrams, preferably less than 25 micrograms, per kg body weight per day). The invention also relates to methods for inhibiting the expression of a target gene, as well as methods for treating diseases caused by the expression of the gene.

    摘要翻译： 本发明涉及具有与靶基因的至少一部分基本上相同且长度不超过49个，优选小于25个核苷酸的核苷酸序列的双链核糖核酸（dsRNA），其中 包含在3'末端具有1至4个核苷酸突出端和5'-末端的互补（反义）RNA链。 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。 药物组合物可用于抑制靶基因的表达，以及用于治疗由靶基因表达引起的疾病，以低剂量（即，小于5毫克，优选小于25微克，每kg体重/ 天）。 本发明还涉及抑制靶基因表达的方法，以及用于治疗由基因表达引起的疾病的方法。