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    Compositions and methods for modulation of IKBKAP splicing
    7.
    发明授权
    Compositions and methods for modulation of IKBKAP splicing 有权

    公开(公告)号：US11066668B2

    公开(公告)日：2021-07-20

    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.

    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
    用于IKBKAP分离调制的组合物和方法

    公开(公告)号：US20150025231A1

    公开(公告)日：2015-01-22

    申请号：US14371941

    申请日：2013-01-11

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

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

    IPC: C12N15/113

    CPC classification number: C12N15/113 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/113 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3233 ,  C12N2310/3521 ,  C12N2310/3525 ,  C12N2310/3533 ,  C12N2320/33

    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.

    Abstract translation: 本公开提供了包含与IKBKAP基因的一部分互补的寡核苷酸的化合物。 某些这样的化合物可用于与IKBKAP基因的一部分杂交，包括但不限于细胞中IKBKAP基因的一部分。 在某些实施方案中，这种杂交导致IKBKAP基因剪接的调节。 在某些实施方案中，IKBKAP基因包括导致有缺陷的剪接和截短的IKAP蛋白的突变。 在某些实施方案中，与IKBKAP基因的一部分互补的寡核苷酸的杂交导致有缺陷的剪接和截短的IKAP蛋白的量的减少。 在某些实施方案中，与IKBKAP基因的一部分互补的寡核苷酸的杂交导致正常剪接和功能性全长IKAP蛋白的量的增加。 在某些实施方案中，寡核苷酸用于治疗家族性生理压力。

    COMPOSITIONS AND METHODS FOR MODULATION OF IKBKAP SPLICING
    10.
    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.

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