公开(公告)号：US10590412B2

公开(公告)日：2020-03-17

申请号：US14785411

申请日：2014-04-21

申请人： Ionis Pharmaceuticals, Inc.

发明人： Amanda Ward ,  Frank Rigo

IPC分类号： C12N15/113

摘要： Disclosed herein are compounds, compositions and methods for modulating the amount or activity of a target nucleic acid. In certain embodiments, the amount or activity of a target nucleic acid is modulated through nonsense mediated decay.

公开(公告)号：US20170362592A1

公开(公告)日：2017-12-21

申请号：US15529428

申请日：2015-11-25

申请人： Ionis Pharmaceuticals, Inc.

发明人： Amanda Ward ,  Frank Rigo

IPC分类号： C12N15/113

CPC分类号： C12N15/1137 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3525 ,  C12Y603/02019

摘要： Certain embodiments are directed to methods and compounds for inhibiting UBE3A-ATS, the endogenous antisense transcript of ubiquitin protein ligase E3A (UBE3A). Such methods and compounds are useful for. Several embodiments provided herein relate to the discovery that antisense compounds targeting UBE3A-ATS induce paternal expression of UBE3A. Several embodiments are drawn to methods and compounds for inducing paternal expression of UBE3A using antisense compounds targeting UBE3A-ATS within a region of UBE3A-ATS that includes the sequence of the small nucleolar RNA (snoRNA), HBII-85 (also referred to as SNORD116).

公开(公告)号：US09617539B2

公开(公告)日：2017-04-11

申请号：US14409348

申请日：2013-06-25

申请人： Ionis Pharmaceuticals, Inc. ,  Baylor College of Medicine

发明人： Frank Rigo ,  Amanda Ward ,  Linyan Meng ,  Arthur L. Beaudet

IPC分类号： C12N15/11 ,  C12N15/113 ,  C07H21/04

CPC分类号： C12N15/113 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/113 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/323 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2320/30 ,  C12N2330/10 ,  C12N2310/3525

摘要： Certain embodiments are directed to methods and compounds for inhibiting UBE3A-ATS, the endogenous antisense transcript of ubiquitin protein ligase E3A (UBE3A). Such methods and compounds are useful for inducing expression of paternal UBE3A in cells and animals.