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    COMPOSITIONS AND METHODS FOR MODULATION OF IKBKAP SPLICING
    8.
    发明申请
    COMPOSITIONS AND METHODS FOR MODULATION OF IKBKAP SPLICING 审中-公开

    公开(公告)号：US20200032260A1

    公开(公告)日：2020-01-30

    申请号：US16422128

    申请日：2019-05-24

    Applicant: Ionis Pharmaceuticals, Inc. ,  Cold Spring Harbor Laboratory

    Inventor： C. Frank Bennett ,  Frank Rigo ,  Adrian R. Krainer ,  Rahul Sinha

    IPC: C12N15/113 ,  C12N15/11

    Abstract: The present disclosure provides compounds comprising oligonucleotides complementary to a portion of the IKBKAP gene. Certain such compounds are useful for hybridizing to a portion of the IKBKAP gene, including but not limited to a portion of the IKBKAP gene in a cell. In certain embodiments, such hybridization results in modulation of splicing of the IKBKAP gene. In certain embodiments, the IKBKAP gene includes a mutation that results in defective splicing and a truncated IKAP protein. In certain embodiments, hybridization of oligonucleotides complementary to a portion of the IKBKAP gene results in a decrease in the amount of defective splicing and truncated IKAP protein. In certain embodiments, hybridization of oligonucleotides complementary to a portion of the IKBKAP gene results in an increase in the amount of normal splicing and functional, full-length IKAP protein. In certain embodiments, oligonucleotides are used to treat Familial Dysautonomia.

    ANTISENSE COMPOUNDS AND USES THEREOF
    10.
    发明申请
    ANTISENSE COMPOUNDS AND USES THEREOF 审中-公开
    抗体化合物及其用途

    公开(公告)号：US20150275211A1

    公开(公告)日：2015-10-01

    申请号：US14440018

    申请日：2013-10-31

    Applicant: ISIS PHARMACEUTICALS, INC. ,  COLD SPRING HARBOR LABORATORY

    Inventor： Frank Rigo ,  C. Bennett ,  Adrian R. Krainer ,  Zhenxun Wang

    IPC: C12N15/113

    CPC classification number: C12N15/113 ,  C12N15/1137 ,  C12N2310/11 ,  C12N2310/14 ,  C12Y207/0104

    Abstract: The present invention provides compounds comprising oligonucleotides complementary to a pyruvate kinase M transcript. Certain such compounds are useful for hybridizing to a pyruvate kinase M transcript, including but not limited to a pyruvate kinase M transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the pyruvate kinase M transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with cancer.

    Abstract translation: 本发明提供了包含与丙酮酸激酶M转录物互补的寡核苷酸的化合物。 某些这样的化合物可用于与丙酮酸激酶M转录物杂交，包括但不限于细胞中的丙酮酸激酶M转录物。 在某些实施方案中，这种杂交导致丙酮酸激酶M转录物剪接的调节。 在某些实施方案中，这些化合物用于治疗与癌症相关的一种或多种症状。

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