- 专利标题： METHOD FOR EFFICIENT EXON (44) SKIPPING IN DUCHENNE MUSCULAR DYSTROPHY AND ASSOCIATED MEANS
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申请号： EP21160161.2申请日： 2009-05-14
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公开(公告)号： EP3872178A1公开(公告)日： 2021-09-01
- 发明人: Platenburg, Gerard Johannes , de Kimpe, Josephus Johannes , van Deutekom, Judith Christina Theodora , van Ommen, Garrit-Jan Boudewijn , Aartsma-Rus, Annemieke
- 申请人： BioMarin Technologies B.V. , Academisch Ziekenhuis Leiden
- 申请人地址： NL 2333 CH Leiden J.H. Oortweg 21; NL 2333 ZA Leiden Albinusdreef 2
- 代理机构： Elend, Almut Susanne
- 优先权： US128010 P 20080515
- 主分类号： C12N15/11
- IPC分类号： C12N15/11 ; A61K31/7088 ; C12N15/86
摘要：
The invention relates to a nucleic acid molecule that binds and/or is complementary to the nucleotide molecule having sequence 5'-GUGGCUAACAGAAGCU (SEQ ID NO 1) and to its use in a method for inducing skipping of exon 44 of the DMD gene in a DMD patient.
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