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.

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.

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：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.