发明名称 Antisense compounds inducing exon skipping and compositions comprising same for treating muscular dystrophy
摘要 The present invention relates to an oligonucleotide which specifically hybridizes to a target region in an exon of the human dystrophin gene inducing exon skipping. The present invention further relates to a pharmaceutical composition comprising an oligonucleotide which specifically hybridizes to a target region in an exon of the human dystrophin gene inducing exon skipping.
申请公布号 IL212386(A) 申请公布日期 2016.10.31
申请号 IL20110212386 申请日期 2011.04.14
申请人 SAREPTA THERAPEUTICS INC. 发明人
分类号 A61K;A61P;C12N 主分类号 A61K
代理机构 代理人
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