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公开(公告)号：US20150025231A1
公开(公告)日：2015-01-22
申请号：US14371941
申请日：2013-01-11
发明人： C. Frank Bennett , Frank Rigo , Adrian R. Krainer , Rahul Sinha
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/11 , C12N2310/113 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3233 , C12N2310/3521 , C12N2310/3525 , C12N2310/3533 , C12N2320/33
摘要： 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.
摘要翻译： 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。
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公开(公告)号：US20200032260A1
公开(公告)日：2020-01-30
申请号：US16422128
申请日：2019-05-24
发明人： C. Frank Bennett , Frank Rigo , Adrian R. Krainer , Rahul Sinha
IPC分类号： C12N15/113 , C12N15/11
摘要： 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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公开(公告)号：US20180371457A1
公开(公告)日：2018-12-27
申请号：US15993090
申请日：2018-05-30
发明人： C. Frank Bennett , Frank Rigo , Adrian R. Krainer , Rahul Sinha
IPC分类号： C12N15/113 , C12N15/11
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/11 , C12N2310/113 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3233 , C12N2310/3521 , C12N2310/3525 , C12N2310/3533 , C12N2320/33
摘要： 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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公开(公告)号：US10344282B2
公开(公告)日：2019-07-09
申请号：US15993090
申请日：2018-05-30
发明人： C. Frank Bennett , Frank Rigo , Adrian R. Krainer , Rahul Sinha
IPC分类号： C12N15/11 , C12N15/113
摘要： 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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公开(公告)号：US11066668B2
公开(公告)日：2021-07-20
申请号：US16422128
申请日：2019-05-24
发明人： C. Frank Bennett , Frank Rigo , Adrian R. Krainer , Rahul Sinha
IPC分类号： C12N15/113 , C12N15/11
摘要： 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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