发明名称 Duchenne型筋ジストロフィーmRNA前駆体のエクソン45の効率的なスキッピングのための方法及び手段
摘要 <p>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 said cell with a molecule that binds to a continuous stretch of at least 21 nucleotides within said exon.</p>
申请公布号 JP5786109(B2) 申请公布日期 2015.09.30
申请号 JP20110533126 申请日期 2009.01.13
申请人 发明人
分类号 C12N15/09;A61K31/7088;A61K35/76;A61K45/00;A61K48/00;A61P21/04;C12N15/113 主分类号 C12N15/09
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