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METHODS FOR MODULATING RNA SPLICING
专利权人:
PTC Therapeutics, Inc.
发明人:
NARYSHKIN, Nikolai,DAKKA, Amal
申请号:
EP20160804178
公开号:
EP3310169(A1)
申请日:
2016.05.27
申请国别(地区):
欧洲专利局
年份:
2018
代理人:
摘要:
In one aspect, described herein is a recognition element for splicing modifier (REMS) that can be recognized by a compound provided herein. In another aspect, described herein are methods for modulating the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene contains a REMS, and the methods utilizing a compound described herein. More particularly, described herein are methods for modulating the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene comprises a REMS, and the methods utilizing a compound described herein. In another aspect, provided herein are artificial gene constructs comprising a REMS, and uses of those artificial gene constructs to modulate functional protein production. In another aspect, provided herein are methods for altering endogenous genes to comprise a REMS, and the use of a compound described herein to modulate the functional protein produced from such altered endogenous
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/

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