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：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.

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.

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。