专利名称 | Oligonucleotide comprising an inosine for treating DMD | ||
申请号 | US15468239 | 申请日 | |
公开(公告)号 | US10533171B2 | 公开(公告)日 | |
申请(专利权)人 | BioMarin Technologies B V | 发明人 | Judith Christina Theodora Van Deutekom; Josephus Johannes De Kimpe; Gerard Johannes Platenburg |
专利来源 | 国家知识产权局 | 转化方式 | |
摘要 |
The invention provides an oligonucleotide comprising an inosine, and/or a nucleotide containing a base able to form a wobble base pair or a functional equivalent thereof, wherein the oligonucleotide, or a functional equivalent thereof, comprises a sequence which is complementary to at least part of a dystrophin pre-m RNA exon or at least part of a non-exon region of a dystrophin pre-m RNA said part being a contiguous stretch comprising at least 8 nucleotides. The invention further provides the use of said oligonucleotide for preventing or treating DMD or BMD. |
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