-
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.
-
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.
-
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。
-
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.
-
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.
-
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.
-
公开(公告)号:US20160298121A1
公开(公告)日:2016-10-13
申请号:US14874420
申请日:2015-10-03
Applicant: Cold Spring Harbor Laboratory
Inventor: Adrian Krainer , Isabel Aznarez
IPC: C12N15/113 , C12Q1/68
CPC classification number: 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
Abstract: Provided herein are methods and compositions for increasing production of a target protein or functional RNA by a cell.
Abstract translation: 本文提供了用于增加细胞产生靶蛋白或功能性RNA的方法和组合物。
-
公开(公告)号:US20210108208A1
公开(公告)日:2021-04-15
申请号:US16878409
申请日:2020-05-19
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.
-
公开(公告)号:US09976143B2
公开(公告)日:2018-05-22
申请号:US14874420
申请日:2015-10-03
Applicant: Cold Spring Harbor Laboratory
Inventor: Adrian Krainer , Isabel Aznarez
IPC: A61K48/00 , C12N15/113 , C07K14/805 , C07K14/47 , C12Q1/68 , C12N15/11
CPC classification number: 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
Abstract: Provided herein are methods and compositions for increasing production of a target protein or functional RNA by a cell.
-
公开(公告)号:US20170159049A9
公开(公告)日:2017-06-08
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.
-
-
-
-
-
-
-
-
-