- 专利标题: Induction of exon skipping in eukaryotic cells
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申请号: EP13170245.8申请日: 2001-09-21
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公开(公告)号: EP2636741A1公开(公告)日: 2013-09-11
- 发明人: van Ommen, Garrit-Jan Boudewijn , van Deutekom, Judith Christina Theodora , Den Dunnen, Johannes Theodorus
- 申请人: Academisch Ziekenhuis Leiden
- 申请人地址: Albinusdreef 2 2333 ZA Leiden NL
- 专利权人: Academisch Ziekenhuis Leiden
- 当前专利权人: Academisch Ziekenhuis Leiden
- 当前专利权人地址: Albinusdreef 2 2333 ZA Leiden NL
- 代理机构: Nederlandsch Octrooibureau
- 优先权: EP00203283 20000921
- 主分类号: C12N15/113
- IPC分类号: C12N15/113 ; A61K48/00 ; A61K31/7088
摘要:
The present invention provides a method for at least in part decreasing the production of an aberrant protein in a cell, said cell comprising pre-mRNA comprising exons coding for said protein, by inducing so-called exon skipping in said cell. Exon-skipping results in mature mRNA that does not contain the skipped exon which leads to an altered product if said exon codes for amino acids. Exon skipping is performed by providing a cell with an agent capable of specifically inhibiting an exon inclusion signal, for instance an exon recognition sequence, of said exon. Said exon inclusion signal can be interfered with by a nucleic acid comprising complementarity to a part of said exon. Said nucleic acid, which is also herewith provided, can be used for the preparation of a medicament, for instance for the treatment of an inherited disease.
公开/授权文献
- EP2636741B1 Induction of exon skipping in eukaryotic cells 公开/授权日:2016-04-27
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