| 发明名称 |
Methods and means for efficient skipping of exon 45 in Duchenne Muscular Dystrophy pre-mRNA |
| 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 said cell with a molecule that binds to a continuous stretch of at least 21 nucleotides within said exon. |
| 申请公布号 |
SI2607484(T1) |
申请公布日期 |
2016.11.30 |
| 申请号 |
SI20090031381 |
申请日期 |
2009.01.13 |
| 申请人 |
BioMarin Technologies B.V.;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 |
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