Antisense oligomers for treatment of non-sense mediated RNA decay based conditions and diseases

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专利权人:

Inc.;Stoke Therapeutics

发明人:

AZNAREZ, Isabel,JING, Enxuan,KACH, Jacob,VENKATESH, Aditya,SCHARNER, Juergen,TICHO, Baruch,LIAU, Gene

申请号：

AU2018355237

公开号：

AU2018355237A1

申请日:

2018.10.23

申请国别(地区):

AU

年份:

2020

代理人:

摘要：

Alternative splicing events in genes can lead to non-productive mRNA transcripts which in turn can lead to aberrant protein expression, and therapeutic agents which can target the alternative splicing events in genes can modulate the expression level of functional proteins in patients and/or inhibit aberrant protein expression. Such therapeutic agents can be used to treat a condition or disease caused by protein deficiency.

来源网站：

中国工程科技知识中心

来源网址：

http://www.ckcest.cn/home/