| 发明名称 |
METHODS AND MEANS FOR EFFICIENT SKIPPING OF EXON 45 IN DUCHENNE MUSCULAR DYSTROPHY PRE-MRNA |
| 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 |
IL212508(D0) |
申请公布日期 |
2011.06.30 |
| 申请号 |
IL20110212508 |
申请日期 |
2011.04.27 |
| 申请人 |
ACADEMISCH ZIEKENHUIS LEIDEN;PROSENSA TECHNOLOGIES B.V.;JUDITH CHRISTINA THEODORA VAN DEUTEKOM;ANNEMIEKE AARTSMA-RUS;JOSEPHUS JOHANNES DE KIMPE;GARRIT-JAN BOUDEWIJN VAN OMMEN;PROSENSA B.V.;PROSENSA HOLDING B.V.;GERADUS JOHANNES PLATENBURG |
发明人 |
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| 分类号 |
C12N |
主分类号 |
C12N |
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