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公开(公告)号：US20220118000A1
公开(公告)日：2022-04-21
申请号：US17379793
申请日：2021-07-19
发明人： Isabel Aznarez , Huw M. Nash , Adrian Krainer
IPC分类号： A61K31/7125 , A61K47/68 , C12N15/113 , A61P43/00 , A61K38/00 , A61K38/17 , C12N15/62 , C12N15/11
摘要： Provided herein are methods and compositions for increasing the expression of a protein, and for treating a subject in need thereof, e.g., a subject with deficient protein expression or a subject having a disease described herein.
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2.发明申请公开(公告)号：US20220088058A1
公开(公告)日：2022-03-24
申请号：US17356147
申请日：2021-06-23
发明人： Isabel Aznarez , Huw M. Nash , Adrian Krainer
IPC分类号： A61K31/7125 , A61K31/712 , C12Q1/6883 , C12N15/113 , C12Q1/68 , A61K31/713 , A61P43/00
摘要： Provided herein are methods and compositions for treating a subject in need thereof, such as a subject with deficient SYNGAP1 protein or SCN1A protein expression or a subject having AD mental retardation 5 or Dravet syndrome.
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3.发明授权公开(公告)号：US11083745B2
公开(公告)日：2021-08-10
申请号：US16062286
申请日：2016-12-14
发明人： Isabel Aznarez , Huw M. Nash , Adrian Krainer
IPC分类号： C07H21/04 , A61K31/7125 , A61K31/712 , C12Q1/6883 , C12N15/113 , C12Q1/68 , A61K31/713 , A61P43/00
摘要： Provided herein are methods and compositions for treating a subject in need thereof, such as a subject with deficient SYNGAP1 protein or SCN1A protein expression or a subject having AD mental retardation 5 or Dravet syndrome.
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公开(公告)号：US20200283762A1
公开(公告)日：2020-09-10
申请号：US16839034
申请日：2020-04-02
发明人： Adrian Krainer , Isabel Aznarez , Tomoki Nomakuchi
IPC分类号： C12N15/11 , A61K31/713 , C12Q1/6883 , A61K31/4245 , A61K31/726
摘要： The present disclosure relates to compositions and methods for inhibiting nonsense-mediated mRNA decay in a gene-specific manner, for example in the treatment of diseases or disorders caused by nonsense mutations.
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公开(公告)号：US10696969B2
公开(公告)日：2020-06-30
申请号：US15949902
申请日：2018-04-10
发明人： Adrian Krainer , Isabel Aznarez
IPC分类号： C12N15/113 , C07K14/805 , C07K14/47 , C12N15/11 , C12Q1/6883
摘要： Provided herein are methods and compositions for increasing production of a target protein or functional RNA by a cell.
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公开(公告)号：US10647983B2
公开(公告)日：2020-05-12
申请号：US14916561
申请日：2014-09-04
发明人： Adrian Krainer , Isabel Aznarez , Tomoki Nomakuchi
IPC分类号： C12N15/11 , A61K31/713 , C12Q1/6883 , A61K31/4245 , A61K31/726 , C07H21/02
摘要： The present disclosure relates to compositions and methods for inhibiting nonsense-mediated mRNA decay in a gene-specific manner, for example in the treatment of diseases or disorders caused by nonsense mutations.
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公开(公告)号：US20160194630A1
公开(公告)日：2016-07-07
申请号：US14916561
申请日：2014-09-04
发明人： Adrian Krainer , Isabel Aznarez , Tomoki Nomakuchi
IPC分类号： C12N15/11 , A61K31/726 , A61K31/4245 , A61K31/713
CPC分类号： C12N15/111 , A61K31/4245 , A61K31/713 , A61K31/726 , C12N2310/11 , C12N2320/30 , C12N2320/31 , C12Q1/6883 , C12Q2600/106 , C12Q2600/156
摘要： The present disclosure relates to compositions and methods for inhibiting nonsense-mediated mRNA decay in a gene-specific manner, for example in the treatment of diseases or disorders caused by nonsense mutations.
摘要翻译： 本公开涉及用于以基因特异性方式抑制无义介导的mRNA衰变的组合物和方法，例如用于治疗由无义突变引起的疾病或病症。
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公开(公告)号：US20210108208A1
公开(公告)日：2021-04-15
申请号：US16878409
申请日：2020-05-19
发明人： Adrian Krainer , Isabel Aznarez
IPC分类号： C12N15/113 , C07K14/805 , C07K14/47 , C12N15/11 , C12Q1/6883
摘要： Provided herein are methods and compositions for increasing production of a target protein or functional RNA by a cell.
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公开(公告)号：US09976143B2
公开(公告)日：2018-05-22
申请号：US14874420
申请日：2015-10-03
发明人： Adrian Krainer , Isabel Aznarez
IPC分类号： A61K48/00 , C12N15/113 , C07K14/805 , C07K14/47 , C12Q1/68 , C12N15/11
CPC分类号： C12N15/1137 , C07K14/47 , C07K14/4702 , C07K14/4736 , C07K14/805 , C12N15/111 , C12N15/113 , C12N2310/11 , C12N2310/315 , C12N2310/3341 , C12N2320/11 , C12N2320/33 , C12Q1/6883 , C12Q2600/136 , C12Q2600/158 , C12Y101/01205 , C12Y304/24087 , C12N2310/321 , C12N2310/3521
摘要： Provided herein are methods and compositions for increasing production of a target protein or functional RNA by a cell.
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公开(公告)号：US20170159049A9
公开(公告)日：2017-06-08
申请号：US14916561
申请日：2014-09-04
发明人： Adrian Krainer , Isabel Aznarez , Tomoki Nomakuchi
IPC分类号： C12N15/11 , A61K31/726 , A61K31/4245 , A61K31/713
CPC分类号： C12N15/111 , A61K31/4245 , A61K31/713 , A61K31/726 , C12N2310/11 , C12N2320/30 , C12N2320/31 , C12Q1/6883 , C12Q2600/106 , C12Q2600/156
摘要： The present disclosure relates to compositions and methods for inhibiting nonsense-mediated mRNA decay in a gene-specific manner, for example in the treatment of diseases or disorders caused by nonsense mutations.
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