BHATTACHARYYA, ANURADHA,DAKKA, AMAL,EFFENBERGER, KERSTIN,GABBETA, VIJAYALAKSHMI,LI, WENCHENG,NARYSHKIN, NIKOLAI,TROTTA, CHRISTOPHER,WIEDINGER, KARI
申请号：
CA3043755
公开号：
CA3043755A1
申请日:
2017.11.27
申请国别(地区):
CA
年份:
2018
代理人:
摘要：
In one aspect, described herein is an intronic recognition element for splicing modifier (iREMS) 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 an intronic 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 an intronic REMS, and the methods utilizing a compound described herein. In another aspect, provided herein are artificial gene constructs comprising an intronic REMS, and uses of those artificial gene constructs to modulate protein production. In another aspect, provided herein are methods for altering endogenous genes to comprise an intronic REMS, and the use of a compound described herein to modulate protein produced from such altered endogenous genes.
