| 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 |
PT2344637(E) |
申请公布日期 |
2015.03.23 |
| 申请号 |
PT20090788160T |
申请日期 |
2009.01.13 |
| 申请人 |
ACADEMISCH ZIEKENHUIS LEIDEN;PROSENSA TECHNOLOGIES B.V.;PROSENSA B.V.;PROSENSA HOLDING BV |
发明人 |
GARRIT-JAN BOUDEWIJN VAN OMMEN;GERARDUS JOHANNES PLATENBURG;JOSEPHUS JOHANNES DE KIMPE;JUDITH CHRISTINA THEODORA VAN DEUTEKOM;ANNEMIEKE AARTSMA-RUS |
| 分类号 |
C12N15/113;A61K31/7105 |
主分类号 |
C12N15/113 |
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