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PROCEDES DE MODIFICATION DE L'EPISSAGE DE L'ARN
专利权人:
PTC THERAPEUTICS; INC.
发明人:
BHATTACHARYYA, ANURADHA,DAKKA, AMAL,EFFENBERGER, KERSTIN,GABBETA, VIJAYALAKSHMI,JANI, MINAKSHI B.,LI, WENCHENG,NARYSHKIN, NIKOLAI,TROTTA, CHRISTOPHER,WIEDINGER, KARI
申请号:
CA3065547
公开号:
CA3065547A1
申请日:
2018.06.13
申请国别(地区):
CA
年份:
2018
代理人:
摘要:
Described herein is an intronic recognition element for splicing modifier (iREMS) that can be recognized by a small molecule splicing modifier compound of Formula (I) provided herein or a form thereof, wherein W, X, A and B are as defined herein. In one aspect, methods for modifying RNA splicing to modulate the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene that contains an intronic REMS is modified utilizing a splicing modifier compound of Formula (I), are described herein. In another aspect, methods for modifying RNA splicing to modulate the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene is modified to comprise an intronic REMS utilizing a splicing modifier compound of Formula (I), are described herein.
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