| 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 a method wherein a molecule is used for inducing and/or promoting skipping of at least one of exon 43, exon 46, exons 50-53 of the DMD pre- mRNA in a patient, preferably in an isolated cell of a patient, the method comprising providing said cell and/or said patient with a molecule. The invention also relates to said molecule as such.</p> |
| 申请公布号 |
EP2344637(A1) |
申请公布日期 |
2011.07.20 |
| 申请号 |
EP20090788160 |
申请日期 |
2009.01.13 |
| 申请人 |
PROSENSA TECHNOLOGIES B.V.;PROSENSA B.V.;PROSENSA HOLDING BV;ACADEMISCH ZIEKENHUIS LEIDEN |
发明人 |
DE KIMPE, JOSEPHUS JOHANNES;PLATENBURG, GERARDUS JOHANNES;VAN DEUTEKOM, JUDITH CHRISTINA THEODORA;AARTSMA-RUS, ANNEMIEKE;VAN OMMEN, GARRIT-JAN BOUDEWIJN |
| 分类号 |
C12N15/113;A61K31/7105 |
主分类号 |
C12N15/113 |
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