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专利概况
专利名称 COMPOSITIONS AND METHODS FOR THE DELIVERY OF mRNA TO HEPATIC CELLS
申请号 WOEP18076667 申请日
公开(公告)号 WO2019063843A1 公开(公告)日
申请(专利权)人 NLIFE THERAPEUTICS S L 发明人 MONTEFELTRO Andr閟 Pablo; REVILLA SANCHEZ Raquel
专利来源 国家知识产权局 转化方式
摘要

The invention provides a composition for selective delivery of an m RNA to a liver cell comprising (i) an mRNA of interest and (ii) one or more conjugates comprising a nucleic acid at least in part substantially complementary to a region of the mRNA, and at least one liver targeting moiety attached to the nucleic acid. The liver targeting moiety comprises at least one selective agent that binds specifically to one or more proteins on the surface of a liver cell. The compounds of the present invention are useful for the delivery of the mRNA of interest to a liver cell of interests where it can be translated into its encoded protein. Thus, the compound of the invention is useful for the treatment of diseases which require the expression of the protein encoded by the mRNA of interest in the target liver cell.

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