申请人:Daiichi Sankyo Company, Limited
公开号:EP3816286A1
公开(公告)日:2021-05-05
The present invention establishes a method for treating cardiac dysfunction. An oligonucleotide of 15-30 bp comprising a nucleotide sequence complementary to a part of the intron 55 region of a dystrophin gene, which comprises the sequence of 5'-TGTCTTCCT-3' or 5'-CAGCTTGAACCGGGC-3' (SEQ ID NO: 64) (wherein "T" may be "U" in either sequence), a pharmacologically acceptable salt thereof, or a solvate thereof. A prophylactic and/or a therapeutic for cardiac dysfunction, comprising the above-described oligonucleotide, a pharmacologically acceptable salt thereof, or a solvate thereof. A suppressor of Dp116 expression, comprising the above-described oligonucleotide, a pharmacologically acceptable salt thereof, or a solvate thereof.
本发明建立了一种治疗心脏功能障碍的方法。一种15-30bp的寡核苷酸,其包含与肌营养不良基因内含子55区部分互补的核苷酸序列,该序列包括5'-TGTCTTCCT-3'或5'-CAGCTTGAACCGGGC-3'(SEQ ID NO:64)(其中 "T "在任一序列中均可为 "U")、其药理学上可接受的盐或其溶液。一种预防和/或治疗心功能不全的药物,包括上述寡核苷酸、其药理学上可接受的盐或其溶液。一种Dp116表达抑制剂,包括上述寡核苷酸、其药理学上可接受的盐或其溶液。