发明名称 ANTISENSE NUCLEIC ACIDS
摘要 The present invention provides a pharmaceutical agent which causes skipping of the 55th, 45th, 50th or 44th exon in the human dystrophin gene with a high efficiency.;The present invention provides an oligomer which efficiently enables to cause skipping of the 55th, 45th, 50th or 44th exon in the human dystrophin gene.
申请公布号 US2014343266(A1) 申请公布日期 2014.11.20
申请号 US201214368307 申请日期 2012.12.27
申请人 NIPPON SHINYAKU CO., LTD. ;NATIONAL CENTER OF NEUROLOGY AND PSYCHIATRY 发明人 Watanabe Naoki;Seo Haruna;Takeda Shin'ichi;Nagata Tetsuya
分类号 C12N15/113 主分类号 C12N15/113
代理机构 代理人
主权项 1. An antisense oligomer which causes skipping of the 55th exon in the human dystrophin gene, consisting of a nucleotide sequence complementary to any one of the nucleotide sequences consisting of the 14th to the 34th, the −2nd to the 19th, the −2nd to the 20th, the −2nd to the 21st, the −2nd to the 22nd, the −2nd to the 23rd, the −1st to the 19th, the −1st to the 20th, the −1st to the 21st, the −1st to the 22nd, the −1st to the 23rd, the 1st to the 19th, the 1st to the 20th, the 1st to the 21st, the 1st to the 22nd, the 1st to the 23rd, the 2nd to the 19th, the 2nd to the 20th, the 2nd to the 21st, the 2nd to the 22nd, the 2nd to the 23rd, the 3rd to the 19th, the 3rd to the 20th, the 3rd to the 21st, the 3rd to the 22nd, the 3rd to the 23rd, the 9th to the 29th, the 9th to the 30th, the 9th to the 31st, the 9th to the 32nd, the 9th to the 33rd, the 10th to the 29th, the 10th to the 30th, the 10th to the 31st, the 10th to the 32nd, the 10th to the 33rd, the 11th to the 29th, the 11th to the 30th, the 11th to the 31st, the 11th to the 32nd, the 11th to the 33rd, the 12th to the 29th, the 12th to the 30th, the 12th to the 31st, the 12th to the 32nd, the 12th to the 33rd, the 13th to the 29th, the 13th to the 30th, the 13th to the 31st, the 13th to the 32nd, the 13th to the 33rd, the 12th to the 34th, the 12th to the 35th, the 12th to the 36th, the 13th to the 34th, the 13th to the 35th, the 13th to the 36th, the 14th to the 32nd, the 14th to the 33rd, 14th to the 35th, the 14th to the 36th, the 15th to the 32nd, the 15th to the 33rd, the 15th to the 34th, the 15th to the 35th, the 15th to the 36th, the 16th to the 32nd, the 16th to the 33rd, the 16th to the 34th, the 16th to the 35th, or the 16th to the 36th nucleotides, from the 5′ end of the 55th exon in the human dystrophin gene.
地址 Kyoto-shi, Kyoto JP