- 专利标题： Methods and means for efficient skipping of exon 45 in Duchenne muscular dystrophy pre-mRNA
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申请号： US13094548申请日： 2011-04-26
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公开(公告)号： US09926557B2公开(公告)日： 2018-03-27
- 发明人: Josephus Johannes De Kimpe , Gerardus Johannes Platenburg , Judith Christina Theodora Van Deutekom , Annemieke Aartsma-Rus , Garrit-Jan Boudewijn Van Ommen
- 申请人： Josephus Johannes De Kimpe , Gerardus Johannes Platenburg , Judith Christina Theodora Van Deutekom , Annemieke Aartsma-Rus , Garrit-Jan Boudewijn Van Ommen
- 申请人地址： NL Leiden NL Leiden
- 专利权人： BioMarin Technologies B.V.,Academisch Ziekenhuis Leiden
- 当前专利权人： BioMarin Technologies B.V.,Academisch Ziekenhuis Leiden
- 当前专利权人地址： NL Leiden NL Leiden
- 代理机构： Jones Day
- 主分类号： C12N15/113
- IPC分类号： C12N15/113 ; A61K31/56 ; A61K31/57 ; A61K38/17 ; A61K31/7088 ; A61K45/06 ; A61K31/573 ; A61K31/58 ; A61K48/00
摘要：
The invention relates to a method for inducing or promoting skipping of exon 45 of DMD pre-mRNA in a Duchenne Muscular Dystrophy patient, preferably in an isolated (muscle) cell, the method comprising providing an isolated muscle cell with a molecule that binds to a continuous stretch of at least 21 nucleotides within said exon. The invention further relates to such molecule used in the method.
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