发明名称 OLIGONUCLEOTIDE FOR THE TREATMENT OF MUSCULAR DYSTROPHY PATIENTS
摘要 The invention relates to an oligonucleotide and to a pharmaceutical composition comprising said oligonucleotide. This oligonucleotide is able to bind to a region of a first exon from a dystrophin pre-mRNA and to a region of a second exon within the same pre-mRNA, wherein said region of said second exon has at least 50% identity with said region of said first exon, wherein said oligonucleotide is suitable for the skipping of said first and second exons of said pre-mRNA, and preferably the entire stretch of exons in between.
申请公布号 HK1209456(A1) 申请公布日期 2016.04.01
申请号 HK20150110181 申请日期 2015.10.16
申请人 PROSENSA TECHNOLOGIES B.V. 发明人 JUDITH CHRISTINA THEODORA VAN DEUTEKOM
分类号 C12N;A61K;A61P;C07H;C12Q 主分类号 C12N
代理机构 代理人
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