- 专利标题: Targeted RNA editing
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申请号: US16807577申请日: 2020-03-03
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公开(公告)号: US11781134B2公开(公告)日: 2023-10-10
- 发明人: Bart Klein , Gerardus Johannes Platenburg
- 申请人: ProQR Therapeutics II B.V.
- 申请人地址: NL Leiden
- 专利权人: ProQR Therapeutics II B.V.
- 当前专利权人: ProQR Therapeutics II B.V.
- 当前专利权人地址: NL Leiden
- 代理机构: STERNE, KESSLER, GOLDSTEIN & FOX P.L.L.C.
- 优先权: GB 22511 2014.12.17 GB 12467 2015.07.16 GB 12595 2015.07.17 GB 21987 2015.12.14
- 主分类号: C12N15/11
- IPC分类号: C12N15/11 ; C12N15/113 ; C12N15/90
摘要:
RNA editing is achieved using oligonucleotide constructs comprising (i) a targeting portion specific for a target nucleic acid sequence to be edited and (ii) a recruiting portion capable of binding and recruiting a nucleic acid editing entity naturally present in the cell. The nucleic acid editing entity, such as ADAR, is redirected to a preselected target site by means of the targeting portion, thereby promoting editing of preselected nucleotide residues in a region of the target RNA which corresponds to the targeting portion.
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