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How to rescue a stop codon through gene reassignment by ACE-tRNA
专利权人:
发明人:
申请号:
JP2020524637
公开号:
JP2021502078A6
申请日:
2018.11.02
申请国别(地区):
JP
年份:
2022
代理人:
摘要:
The present invention is a composition and method for treating diseases and disorders associated with an immature stop codon (PTC), which comprises or encodes an anticodon editing (ACE) tRNA (ACE-tRNA). Provided are methods that include administration of nucleic acid molecules. Specifically, the method comprises administering to the subject at least one ACE-tRNA specific for UGA, UAA, or UAG to treat the associated disorder. In addition, the nucleic acid sequence of ACE-tRNA is disclosed.
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中国工程科技知识中心
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http://www.ckcest.cn/home/

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