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ANTISENSE OLIGOMERS FOR TREATMENT OF CONDITIONS AND DISEASES
专利权人:
Inc.;Stoke Therapeutics
发明人:
Isabel AZNAREZ,Zhou HAN
申请号:
US16561952
公开号:
US20200024603A1
申请日:
2019.09.05
申请国别(地区):
US
年份:
2020
代理人:
摘要:
Alternative splicing events in SCN1A gene 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 SCN1A gene can modulate the expression level of functional proteins in Dravet Syndrome patients and/or inhibit aberrant protein expression. Such therapeutic agents can be used to treat a condition caused by SCN1A, SCN8A or SCN5A 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/
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