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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
Applicant: Stoke Therapeutics, Inc. , Cold Spring Harbor Laboratory
Inventor： Isabel Aznarez , Huw M. Nash , Adrian Krainer
IPC: A61K31/7125 , A61K47/68 , C12N15/113 , A61P43/00 , A61K38/00 , A61K38/17 , C12N15/62 , C12N15/11
Abstract: 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
Applicant: Cold Spring Harbor Laboratory , Stoke Therapeutics, Inc.
Inventor： Isabel Aznarez , Huw M. Nash , Adrian Krainer
IPC: A61K31/7125 , A61K31/712 , C12Q1/6883 , C12N15/113 , C12Q1/68 , A61K31/713 , A61P43/00
Abstract: 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
Applicant: Cold Spring Harbor Laboratory , Stoke Therapeutics, Inc.
Inventor： Isabel Aznarez , Huw M. Nash , Adrian Krainer
IPC: C07H21/04 , A61K31/7125 , A61K31/712 , C12Q1/6883 , C12N15/113 , C12Q1/68 , A61K31/713 , A61P43/00
Abstract: 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
Applicant: Cold Spring Harbor Laboratory
Inventor： Adrian Krainer , Isabel Aznarez , Tomoki Nomakuchi
IPC: C12N15/11 , A61K31/713 , C12Q1/6883 , A61K31/4245 , A61K31/726
Abstract: 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
Applicant: Cold Spring Harbor Laboratory
Inventor： Adrian Krainer , Isabel Aznarez
IPC: C12N15/113 , C07K14/805 , C07K14/47 , C12N15/11 , C12Q1/6883
Abstract: Provided herein are methods and compositions for increasing production of a target protein or functional RNA by a cell.
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Patent Agency Ranking
公开(公告)号：US10647983B2
公开(公告)日：2020-05-12
申请号：US14916561
申请日：2014-09-04
Applicant: Cold Spring Harbor Laboratory
Inventor： Adrian Krainer , Isabel Aznarez , Tomoki Nomakuchi
IPC: C12N15/11 , A61K31/713 , C12Q1/6883 , A61K31/4245 , A61K31/726 , C07H21/02
Abstract: 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
Applicant: COLD SPRING HARBOR LABORATORY
Inventor： Adrian Krainer , Isabel Aznarez , Tomoki Nomakuchi
IPC: C12N15/11 , A61K31/726 , A61K31/4245 , A61K31/713
CPC classification number: C12N15/111 , A61K31/4245 , A61K31/713 , A61K31/726 , C12N2310/11 , C12N2320/30 , C12N2320/31 , C12Q1/6883 , C12Q2600/106 , C12Q2600/156
Abstract: 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.
Abstract translation: 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：US11352624B2
公开(公告)日：2022-06-07
申请号：US16839034
申请日：2020-04-02
Applicant: Cold Spring Harbor Laboratory
Inventor： Adrian Krainer , Isabel Aznarez , Tomoki Nomakuchi
IPC: C12N15/11 , A61K31/713 , C12Q1/6883 , A61K31/4245 , A61K31/726
Abstract: 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：US11096956B2
公开(公告)日：2021-08-24
申请号：US16007435
申请日：2018-06-13
Applicant: Stoke Therapeutics, Inc. , Cold Spring Harbor Laboratory
Inventor： Isabel Aznarez , Huw M. Nash , Adrian Krainer
IPC: A61K31/7125 , A61K47/68 , C12N15/113 , A61P43/00 , A61K38/00 , A61K38/17 , C12N15/62 , C12N15/11
Abstract: 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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公开(公告)号：US20180362987A1
公开(公告)日：2018-12-20
申请号：US15949902
申请日：2018-04-10
Applicant: Cold Spring Harbor Laboratory
Inventor： Adrian Krainer , Isabel Aznarez
IPC: C12N15/113 , C07K14/47 , C07K14/805 , C12N15/11 , C12Q1/6883
Abstract: Provided herein are methods and compositions for increasing production of a target protein or functional RNA by a cell.
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