- 专利标题： METHODS AND MEANS FOR EFFICIENT SKIPPING OF AT LEAST ONE OF THE FOLLOWING EXONS OF THE HUMAN DUCHENNE MUSCULAR DYSTROPHY GENE: 43, 46, 50-53
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申请号： US18771603申请日： 2024-07-12
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公开(公告)号： US20240360452A1公开(公告)日： 2024-10-31
- 发明人: Judith Christina Theodora van Deutekom
- 申请人： BioMarin Technologies B.V. , Academisch Ziekenhuis Leiden
- 申请人地址： NL Amsterdam
- 专利权人： BioMarin Technologies B.V.,Academisch Ziekenhuis Leiden
- 当前专利权人： BioMarin Technologies B.V.,Academisch Ziekenhuis Leiden
- 当前专利权人地址： NL Amsterdam
- 主分类号： C12N15/113
- IPC分类号： C12N15/113 ; A61K31/56 ; A61K31/57 ; A61K31/573 ; A61K31/58 ; A61K31/7088 ; A61K38/17 ; A61K45/06 ; A61K48/00
摘要：
The invention relates to a method wherein a molecule is used for inducing and/or promoting skipping of at least one of exon 43, exon 46, or exons 50-53 of the DMD pre-mRNA in a patient, the method comprising providing the patient with the molecule. The invention also relates to the molecule as such.
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