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专利概况
专利名称 RNA PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND USES THEREOF
申请号 WOUS19041051 申请日
公开(公告)号 WO2020014261A1 公开(公告)日
申请(专利权)人 THE BROAD INSTITUTE INC; PRESIDENT AND FELLOWS OF HARVARD COLLEGE 发明人 LIU David R; WILSON Christopher Gerard; CHEN Peter J
专利来源 国家知识产权局 转化方式
摘要

The disclosure provides programmable methylation "writers" and demethylation "erasers" for editing the methylation state of RNA targets, e.g., an RNA transcriptome. In particular, the disclosure provides RNA methylation editor polynucleotide contructs and vectors comprising (i) an RNA programmable RNA binding domain (RNApRNAbd); and (ii) an effector domain, wherein the effector domain is capable of adding or removing a methyl group in an RNA. The disclosed RNA methylation editor constructs are capable of achieving limited off- target modifications in RNA molecules. Further, the disclosure provides methods for making and using the programmable methylation editors to modifying the methylation state of RNA. The disclosure further provides complexes comprising a methylation writer protein and a guide RNA molecule and complexes comprising a demethylation eraser protein and a guide RNA molecule. The disclosure further provides pharmaceutical compositions and cells comprising the disclosed fusion proteins and complexes.

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