Compositions and methods for inhibiting expression of a mutant gene
    11.
    发明授权
    Compositions and methods for inhibiting expression of a mutant gene 有权
    用于抑制突变基因表达的组合物和方法

    公开(公告)号:US08273870B2

    公开(公告)日:2012-09-25

    申请号:US13177316

    申请日:2011-07-06

    IPC分类号: C07H21/04

    摘要: The present invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a mutant gene, comprising a first strand that has a complementary region that is complementary to at least a portion of an RNA transcript of at least part of the mutant target gene and a second strand of the dsRNA complementary or substantially complementary to the first strand. 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 mutant gene, as well as for treating diseases caused by expression of the target gene. The invention also relates to methods for inhibiting the expression of a target mutant gene, as well as methods for treating diseases caused by the expression of the target gene.

    摘要翻译: 本发明涉及用于抑制突变基因表达的双链核糖核酸(dsRNA),其包含第一链,该第一链具有与至少部分至少部分的RNA转录物的至少一部分互补的互补区 突变靶基因和与第一链互补或基本上互补的dsRNA的第二链。 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突变基因的表达,以及用于治疗由靶基因表达引起的疾病。 本发明还涉及抑制靶突变基因表达的方法,以及用于治疗由靶基因表达引起的疾病的方法。

    Compositions and methods for inhibiting expression of a mutant gene
    12.
    发明授权
    Compositions and methods for inhibiting expression of a mutant gene 有权
    用于抑制突变基因表达的组合物和方法

    公开(公告)号:US07994309B2

    公开(公告)日:2011-08-09

    申请号:US12817009

    申请日:2010-06-16

    IPC分类号: C07H21/04

    摘要: The present invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a mutant gene, comprising a complementary RNA strand having a complementary region that is substantially complementary to a portion of the mutant gene, and which is partially complementary to the corresponding wild-type gene. 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 mutant gene, as well as for treating diseases caused by expression of the target gene. The invention also relates to methods for inhibiting the expression of a target mutant gene, as well as methods for treating diseases caused by the expression of the target gene.

    摘要翻译: 本发明涉及用于抑制突变基因表达的双链核糖核酸(dsRNA),其包含互补RNA链,其具有与突变基因的一部分基本上互补的互补区,并且其部分互补于 相应的野生型基因。 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突变基因的表达,以及用于治疗由靶基因表达引起的疾病。 本发明还涉及抑制靶突变基因表达的方法,以及用于治疗由靶基因表达引起的疾病的方法。

    Compositions and methods for inhibiting expression of a mutant gene
    13.
    发明授权
    Compositions and methods for inhibiting expression of a mutant gene 有权
    用于抑制突变基因表达的组合物和方法

    公开(公告)号:US07763590B2

    公开(公告)日:2010-07-27

    申请号:US10384463

    申请日:2003-03-07

    IPC分类号: A61K48/00

    摘要: The present invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a mutant gene, comprising a complementary RNA strand having a complementary region that is substantially complementary to a portion of the mutant gene, and which is partially complementary to the corresponding wild-type gene. 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 mutant gene, as well as for treating diseases caused by expression of the target gene. The invention also relates to methods for inhibiting the expression of a target mutant gene, as well as methods for treating diseases caused by the expression of the target gene.

    摘要翻译: 本发明涉及用于抑制突变基因表达的双链核糖核酸(dsRNA),其包含互补RNA链,其具有与突变基因的一部分基本上互补的互补区,并且其部分互补于 相应的野生型基因。 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突变基因的表达,以及用于治疗由靶基因表达引起的疾病。 本发明还涉及抑制靶突变基因表达的方法,以及用于治疗由靶基因表达引起的疾病的方法。

    Compositions and Methods for Inhibiting Expression of a Mutant Gene
    15.
    发明申请
    Compositions and Methods for Inhibiting Expression of a Mutant Gene 审中-公开
    抑制突变基因表达的组合物和方法

    公开(公告)号:US20120322858A1

    公开(公告)日:2012-12-20

    申请号:US13594140

    申请日:2012-08-24

    摘要: The present invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a mutant gene, comprising a first strand that has a complementary region that is complementary to at least a portion of an RNA transcript of at least part of the mutant target gene and a second strand of the dsRNA complementary or substantially complementary to the first strand. 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 mutant gene, as well as for treating diseases caused by expression of the target gene. The invention also relates to methods for inhibiting the expression of a target mutant gene, as well as methods for treating diseases caused by the expression of the target gene.

    摘要翻译: 本发明涉及用于抑制突变基因表达的双链核糖核酸(dsRNA),其包含第一链,该第一链具有与至少部分至少部分的RNA转录物的至少一部分互补的互补区 突变靶基因和与第一链互补或基本上互补的dsRNA的第二链。 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突变基因的表达,以及用于治疗由靶基因表达引起的疾病。 本发明还涉及抑制靶突变基因表达的方法,以及用于治疗由靶基因表达引起的疾病的方法。

    Compositions and Methods for Inhibiting Expression of a Mutant Gene
    16.
    发明申请
    Compositions and Methods for Inhibiting Expression of a Mutant Gene 有权
    抑制突变基因表达的组合物和方法

    公开(公告)号:US20110287090A1

    公开(公告)日:2011-11-24

    申请号:US13177316

    申请日:2011-07-06

    摘要: The present invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a mutant gene, comprising a first strand that has a complementary region that is complementary to at least a portion of an RNA transcript of at least part of the mutant target gene and a second strand of the dsRNA complementary or substantially complementary to the first strand. 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 mutant gene, as well as for treating diseases caused by expression of the target gene. The invention also relates to methods for inhibiting the expression of a target mutant gene, as well as methods for treating diseases caused by the expression of the target gene.

    摘要翻译: 本发明涉及用于抑制突变基因表达的双链核糖核酸(dsRNA),其包含第一链,该第一链具有与至少部分至少部分的RNA转录物的至少一部分互补的互补区 突变靶基因和与第一链互补或基本上互补的dsRNA的第二链。 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突变基因的表达,以及用于治疗由靶基因表达引起的疾病。 本发明还涉及抑制靶突变基因表达的方法,以及用于治疗由靶基因表达引起的疾病的方法。

    Compositions and Methods for Inhibiting Expression of a Mutant Gene
    17.
    发明申请
    Compositions and Methods for Inhibiting Expression of a Mutant Gene 有权
    抑制突变基因表达的组合物和方法

    公开(公告)号:US20100291194A1

    公开(公告)日:2010-11-18

    申请号:US12817009

    申请日:2010-06-16

    摘要: The present invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a mutant gene, comprising a complementary RNA strand having a complementary region that is substantially complementary to a portion of the mutant gene, and which is partially complementary to the corresponding wild-type gene. 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 mutant gene, as well as for treating diseases caused by expression of the target gene. The invention also relates to methods for inhibiting the expression of a target mutant gene, as well as methods for treating diseases caused by the expression of the target gene.

    摘要翻译: 本发明涉及用于抑制突变基因表达的双链核糖核酸(dsRNA),其包含互补RNA链,其具有与突变基因的一部分基本上互补的互补区,并且其部分互补于 相应的野生型基因。 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突变基因的表达,以及用于治疗由靶基因表达引起的疾病。 本发明还涉及抑制靶突变基因表达的方法,以及用于治疗由靶基因表达引起的疾病的方法。

    Compositions and methods for inhibiting expression of a mutant gene
    18.
    发明申请
    Compositions and methods for inhibiting expression of a mutant gene 有权
    用于抑制突变基因表达的组合物和方法

    公开(公告)号:US20050074757A1

    公开(公告)日:2005-04-07

    申请号:US10384463

    申请日:2003-03-07

    摘要: The present invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a mutant gene, comprising a complementary RNA strand having a complementary region that is substantially complementary to a portion of the mutant gene, and which is partially complementary to the corresponding wild-type gene. 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 mutant gene, as well as for treating diseases caused by expression of the target gene. The invention also relates to methods for inhibiting the expression of a target mutant gene, as well as methods for treating diseases caused by the expression of the target gene.

    摘要翻译: 本发明涉及用于抑制突变基因表达的双链核糖核酸(dsRNA),其包含互补RNA链,其具有与突变基因的一部分基本上互补的互补区,并且其部分互补于 相应的野生型基因。 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突变基因的表达,以及用于治疗由靶基因表达引起的疾病。 本发明还涉及抑制靶突变基因表达的方法,以及用于治疗由靶基因表达引起的疾病的方法。